JP Morgan 2026

Clinical stage biotechnology company discovering and developing allogeneic “off-the-shelf” gamma delta CAR T cell therapies for autoimmune diseases and cancer. Lead candidate ADI-001, a B-cell depleting off-the-shelf gamma-delta CAR T cell therapy, is being evaluated in a Phase 1 study in four autoimmune indications including lupus nephritis (LN), systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV), idiopathic inflammatory myopathy (IIM), and stiff person syndrome (SPS). Clinical update from ADI-001 trial in LN is expected in 1H25, and for other autoimmune diseases in 2H25. ADI-270, a highly differentiated armored off-the-shelf gamma delta CAR T cell therapy candidate targeting CD70-positive cancers, is being investigated in renal cell carcinoma (RCC) and preliminary clinical data planned in 1H25. Granted FDA Fast Track designation for ADI-001 in relapsed/refractory class III or class IV LN and for ADI-270 in metastatic/advanced clear cell RCC patients.

Biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. Arrakis is building a proprietary pipeline of RNA-targeted small molecule (rSM) medicines focused on cancer and genetically validated targets in other disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors.

Arvinas is a clinical-stage biotechnology company dedicated to improving the lives of patients suffering from debilitating and life-threatening diseases. Through its PROTAC (PROteolysis TArgeting Chimera) protein degrader platform, the Company is pioneering the development of protein degradation therapies designed to harness the body’s natural protein disposal system to selectively and efficiently degrade and remove disease-causing proteins. Arvinas is currently progressing multiple investigational drugs through clinical development programs, including vepdegestrant, targeting the estrogen receptor for patients with locally advanced or metastatic ER+/HER2- breast cancer; ARV-393, targeting BCL6 for relapsed/refractory non-Hodgkin Lymphoma; ARV-102, targeting LRRK2 for neurodegenerative disorders; and ARV-806, targeting KRAS G12D for mutated cancers, including pancreatic and colorectal cancers. 

Clinical-stage biotechnology company developing therapies to precisely engage specific immune cells to fight cancer and chronic viral infections utilizing a proprietary cis-targeting platform. Lead candidate AB248, an IL-2 molecule specifically targeted to CD8+ effector T cells, is currently in an ongoing Phase 1a/1b trial for the treatment of locally advanced or metastatic solid tumors. Initial pharmacokinetic and pharmacodynamic data from the ongoing Phase 1a/1b clinical trial support AB248’s proof of mechanism and activity with a highly differentiated clinical profile. Early data shows potent and selective CD8+ T cell activation without substantial changes to Treg and NK cell numbers and initial evidence of anti-tumor activity, with a generally well-tolerated safety profile. Broader portfolio includes AB821, an IND-ready CD8-targeted IL-21 immunotherapy, and early stage programs targeting CAR-T cells, myeloid cells and CD4+ T cells.

Late-stage biopharmaceutical company focused on discovering, developing and commercializing oral therapies to address the unmet medical needs of patients with serious viral infections. Lead candidate, bemnifosbuvir, a nucleotide polymerase inhibitor, is an oral, direct-acting antiviral drug candidate being evaluated in combination with ruzasvir, an oral NS5A inhibitor, in a Phase 2 trial in treatment-naïve, HCV-infected patients either without cirrhosis or with compensated cirrhosis. Patient enrollment for the trial has been completed and initial results are expected in 4Q24. Atea has built a proprietary nucleos(t)ide prodrug platform to develop novel product candidates to treat single stranded ribonucleic acid, or ssRNA, viruses, which are a prevalent cause of serious viral diseases.

Clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration. Athira aims to alter the course of neurological diseases by advancing its pipeline of drug candidates that modulate the neurotrophic HGF system. Advancing ATH-1105, a next-generation, orally administered, orally-delivered, positive modulator of the neurotrophic HGF system that is in development as a treatment for ALS. ATH-1105 is in a Phase 1 first-in-human, dose escalation study in healthy volunteers trial completion expected by year-end 2024. In preclinical models of ALS, ATH-1105 has been shown to significantly increase survival, enhance motor and nerve function, reduce peripheral nerve demyelination and axon degeneration, and improve neurodegeneration and inflammation. The Phase 1 study is expected to complete by year end 2024 and commencement of dosing of ALS patients is expected in 2025.

Avadel Pharmaceuticals plc is a biopharmaceutical company focused on transforming medicines to transform lives. Its commercial product, LUMRYZ™, is the first and only once-at-bedtime, extended-release sodium oxybate approved for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. Backed by strong real-world data and rapid adoption, LUMRYZ generated $120.6M of revenue in the first half of 2025, up 76% year-over-year.  The number of patients on LUMRYZ increased to 3,100 as of June 30, 2025, up 63% versus June 30, 2024. There are currently 31 US patents having expiries ranging from mid-2037 to early-2042 listed in FDA’s Orange Book for LUMRYZ.  Additionally, LUMRYZ was granted seven years of orphan drug exclusivity (ODE) for the treatment of narcolepsy in adults through May 1, 2030 and was also granted ODE in pediatric narcolepsy patients 7 years and older through October 16, 2031.   FDA has also granted orphan drug designation (ODD) to LUMRYZ for the treatment of idiopathic hypersomnia (IH). Avadel is currently conducting its Phase 3 REVITALYZ™ study for LUMRYZ in IH and expects to complete enrollment by year end 2025 with data readout anticipated in the first half of 2026 and a potential NDA filing for IH in second half 2026. Avadel recently expanded its pipeline through an exclusive license for valiloxybate (VOX), an oxybate amino acid conjugate enabling development of a once-at-bedtime, salt-free and artificial sweetener-free oxybate formulation. The company currently plans to initiate pharmacokinetic (PK) studies on its VOX formulation candidate in Q4 2025, and, if successful, initiate the pivotal PK trial in the second half of 2026.

Clinical-stage biopharmaceutical company focused on developing targeted therapies for serious liver disease. Potentially best in class lead asset efimosfermin (BOS-580) is a highly-engineered, long-acting, once monthly subcutaneous injection of human fibroblast growth factor 21 (FGF21) for the treatment of metabolic dysfunction associated steatohepatitis (MASH) and in Phase 1B and 2a has shown statistically significant efficacy across all key liver and selected metabolic biomarkers, after only 3 doses, along with an overall positive side effect profile. Expecting to announce Phase 2 data in F2/F3 patients in 4Q24, with Phase 2 Part C extension data, as well as Part D, non-invasive biomarkers and biopsy data in advanced fibrosis patients, expected in 2025.

Clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies that have the potential to provide a deep and durable, perhaps curative, treatment for patients with autoimmune diseases. The CABA™ platform encompasses two strategies: the CARTA (chimeric antigen receptor T cells for autoimmunity) strategy, with CABA-201, a 4-1BB-containing fully human CD19-CAR T, as the lead product candidate being evaluated in the RESET™ (REstoring SElf-Tolerance) clinical trials in myositis, systemic lupus erythematosus, systemic sclerosis, generalized myasthenia gravis and in the RESET-PV™ sub-study within the DesCAARTes™ clinical trial in pemphigus vulgaris, along with the CAART (chimeric autoantibody receptor T cells) strategy, with multiple clinical-stage candidates, including DSG3-CAART for mucosal pemphigus vulgaris and MuSK-CAART for MuSK-associated myasthenia gravis. The expanding CABA™ platform is designed to develop potentially curative therapies that offer deep and durable responses for patients with a broad range of autoimmune diseases. Cabaletta Bio’s headquarters and labs are located in Philadelphia, PA.

Biopharmaceutical company with a mission to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. The Company’s most advanced clinical-stage development program, ONC201, is in Phase 3 development for H3 K27M-mutant glioma.   The Company is conducting Phase 1 dose escalation studies of ONC206 to evaluate safety and PK data.

Pioneering a novel class of masked, conditionally activated biologics, powered by its PROBODY® platform, with a pipeline across multiple treatment modalities, focused on major unmet needs in oncology. CX-2051, an EpCAM-directed ADC with a Topo-1 payload with potential applicability across multiple EpCAM-expressing epithelial cancers, demonstrated positive data and a favorable safety profile along with encouraging initial signs of efficacy in CRC, anticipated to have data update in 1Q26 and initiate Phase 2 in 1H26. CX-801, an interferon alpha-2b cytokine, with broad potential applicability in traditionally immune-oncology sensitive as well as insensitive tumors, anticipated to have Phase 1a monotherapy data in 4Q25 and combo data in advanced melanoma in 2026. Strategic collaborations with leaders in oncology such as Amgen, Astellas, Bristol Myers Squibb, Regeneron and Moderna. 

Clinical-stage biopharmaceutical company building a portfolio of innovative, first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. Lead therapeutic candidate, bitopertin, is a first-in-class oral inhibitor of GlyT1, which has the potential to be the first disease-modifying treatment for erythropoietic protoporphyria (EPP). Disc has two additional pipeline candidates focused on iron modulation – including DISC-0974, an anti-hemojuvelin (HJV) antibody targeting anemias in myelofibrosis and chronic kidney disease driven by high hepcidin, and DISC-3405 targeting Transmembrane Serine Protease 6 (TMPRSS6) for indications characterized by iron overload and excess red blood cells (polycythemia vera, sickle cell disease). 

Clinical-stage oncology company focused on the discovery and development of selective cancer therapies to treat patients across a range of solid tumors. Currently evaluating EO-3021, an anti-Claudin 18.2 ADC, in a Phase 1 study in patients with advanced, unresectable or metastatic solid tumors likely to express Claudin 18.2, including gastric, gastroesophageal, pancreatic or esophageal cancers. In August 2024, announced promising initial data, demonstrating 42.8% confirmed ORR observed in Claudin 18.2-enriched subset of gastric and GEJ cancer. Advancing into dose expansion portion of ongoing Phase 1 study of EO-3021 and expect additional monotherapy data in 1H 2025. Secured clinical supply agreements to evaluate EO-3021 in combination with ramucirumab and dostarlimab with Lilly and GSK, respectively, and expect to initiate dosing in combination portion by year-end 2024. Also advancing a HER3-targeting ADC for patients with solid tumors that overexpress HER3; expect to nominate a development candidate before year-end.

Commercial-stage global biopharmaceutical company improving outcomes for patients with or at risk for cardiovascular and cardiometabolic diseases.  Esperion is commercializing NEXLETOL and NEXLIZET, the first oral non-statin LDL-C lowering drugs to be approved by the FDA to reduce the risk of CV events in both primary and secondary prevention patients. These medications are supported by the nearly 14,000 patient CLEAR Cardiovascular Outcomes Trial demonstrating their ability to significantly reduced cardiovascular risk and the risk of heart attack and coronary revascularization. The 2025 update of the ESC/EAS Guidelines for the Management of Dyslipidaemias further validates the clinical benefit of bempedoic acid in cardiovascular risk reduction as the only non-statin to receive a Level 1a recommendation. In 2Q 2025, the company achieved its first quarter of operating income from ongoing business, with plans for sustainable profitability starting in 1Q26, and realized a 10% increase in total retail prescription equivalents. Esperion continues to evolve into a leading global biopharmaceutical company through commercial execution, international partnerships, collaborations, and advancement of its pre-clinical pipeline.

EyePoint is committed to developing and commercializing innovative therapeutics to help improve the lives of patients with serious retinal diseases. EyePoint’s pipeline includes DURAVYU™ (f/k/a EYP-1901), an investigational sustained delivery treatment for VEGF-mediated retinal diseases combining vorolanib, a selective and patent-protected tyrosine kinase inhibitor with bioerodible Durasert E™. DURAVYU is currently in Phase 2 clinical trials as a maintenance treatment for wet age-related macular degeneration with first patient dosing in the Phase 3 trials expected in 2024. DURAVYU is also being studied in the Phase 2 VERONA trial for diabetic macular edema with topline data expected in Q1 2025. Additional pipeline programs include EYP-2301, a novel TIE 2 activator being studied as a potential new MOA for treating serious retinal diseases and complement inhibition for geographic atrophy. EyePoint ended Q2 2024 with ~$280 million of cash and equivalents with cash guidance through Phase 3 wet AMD topline data in 2026.

Clinical-stage biotechnology company developing novel vaccines for many of the world’s most threatening infectious diseases and therapies for solid tumor cancers. The Company’s lead clinical program is GEO-CM04S1, a next-generation COVID-19 vaccine for which GeoVax was recently awarded a BARDA-funded contract to sponsor a 10,000-participant Phase 2b clinical trial to evaluate the efficacy of GEO-CM04S1 versus an approved mRNA COVID-19 vaccine. In addition, GEO-CM04S1 is currently in three Phase 2 clinical trials, being evaluated as (1) a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized COVID-19 vaccines are insufficient, (2) a booster vaccine in patients with chronic lymphocytic leukemia (CLL) and (3) a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. Also, GeoVax is advancing GEO-MVA, a vaccine against Mpox/Smallpox through cGMP manufacturing in order to potentially assist in the WHO declared global health emergency.  In oncology the lead clinical program is evaluating a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, having recently completed in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax recently announced plans to initiate a Phase 2 trial of Gedeptin® in combination with an immune checkpoint inhibitor, with that study anticipated to initiate in mid-2025. GeoVax has a strong IP portfolio in support of its technologies and product candidates, holding worldwide rights for its technologies and products. The Company’s leadership team has driven significant value creation across multiple life science companies over the past several decades. GeoVax is headquartered in the metropolitan Atlanta, GA area.

Advanced clinical-stage private company developing Padeliporfin Vascular Targeted Photodynamic (VTP) therapy, delivering non-thermal laser light to locally activate Padeliporfin as a targeted treatment for unresectable solid tumors. Interim Ph. 3 data achieved CR rate of 73% with well-tolerated safety profile in low-grade upper tract urinary cancer (LG-UTUC); topline data expected in 4Q25 and NDA submission anticipated in 1Q26. Additional programs include PDAC (Phase 1); high-grade UTUC (Phase 1 ready) and NSCLC (Phase 1 ready). 

Late clinical-stage biopharmaceutical company developing LNZ100, a preservative-free, single-use, once-daily aceclidine based eye drop, for patients with presbyopia, a condition characterized by inevitable loss of near vision impacting an estimated 128 million people in the United States. In registrational Phase 3 CLARITY study, LNZ100 demonstrated strong tolerability as well as safety profile and showed near vision improvement and statistically significant three-lines or greater improvement in Best Corrected Distance Visual Acuity (BCDVA) at near, without losing one line or more in distance visual acuity. Submitted a New Drug Application (NDA) to the FDA for the treatment of presbyopia with potential for commercial launch as early as 2H2025.

Clinical-stage biopharmaceutical company founded to confront the greatest medical challenges of our generation by taking a fundamentally different approach to the way treatments for brain diseases are developed. Therapeutic pipeline currently consists of seven programs that target novel mechanisms of action for a broad range of underserved, prevalent diseases. Neumora’s mission is to redefine neuroscience drug development by bringing forward the next generation of novel therapies that offer improved treatment outcomes and quality of life for patients. 

NewAmsterdam Pharma is a late-clinical stage biopharmaceutical company developing obicetrapib, a once-daily oral CETP inhibitor for patients with cardiovascular disease who need additional LDL-C lowering beyond statins. Across multiple Phase 3 trials, obicetrapib has demonstrated up to 50% LDL-C reduction when combined with ezetimibe, and 35-40% as monotherapy. With regulatory submissions planned and over $700 million to support development and commercial-readiness, NewAmsterdam is advancing obicetrapib as a potential foundational therapy in lipid management.

Biotechnology company leveraging the extraordinary adaptive biology of bats to unlock new insights and accelerate the discovery and development of innovative therapies for humans, with an initial focus on immunology & inflammation and cardiometabolism. Paratus' proprietary platform integrates cell biology, genomics and informatics alongside its unique expertise in bat biology data and comparative biology to compare bats' evolved patterns of disease resistance to humans' patterns of disease development to address significant challenges across a spectrum of therapeutic areas. Nominated lead ASC development candidate with IND/CTA submission anticipated in 4Q26. Multiple targets advancing through target validation, with the most advanced metabolic target in lead optimization. Backed by top-tier investor syndicate, including Polaris Partners, ARCH Venture Partners, ClavystBio, EcoR1 Capital, Leaps by Bayer, and Alexandria Venture Investments.

Clinical stage biotechnology company focused on generating and developing novel antibody therapeutics selectively targeting toxic misfolded proteins in neurodegenerative diseases such as Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA). Lead candidate, PMN310, is humanized monoclonal antibody designed to selectively bind toxic forms of amyloid beta. ProMIS reported successful top-line data from its first-in-human Phase 1a clinical trial, which showed that PMN310 was safe and well tolerated. The Company expects to initiate a Phase 1b clinical trial in Alzheimer's disease later this year. In July 2024, the Company announced a private placement financing of up to $122.7 million to support its novel antibody therapeutics for AD, ALS and MSA.

Clinical-stage biotechnology company translating scientific advances into transformative therapies for patients with devastating rare diseases. Rallybio has built a broad pipeline of promising product candidates aimed at addressing diseases with unmet medical need in areas of maternal fetal health, complement dysregulation, hematology, and metabolic disorders. Rallybio is advancing two clinical stage programs: RLYB212, an anti-HPA-1a antibody for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT) and RLYB116, an inhibitor of complement component 5 (C5), with the potential to treat several diseases of complement dysregulation. Additional preclinical programs include RLYB332, a long-acting MTP-2 inhibitor for the treatment of diseases of iron overload, an ENPP1 Inhibitor for the treatment of Hypophosphatasia (HPP), and RLYB114, a C5 inhibitor for ophthalmic use.

Clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs and cells implicated in disease, enabling improved efficacy and potency. Lead programs include RCT2100 for the treatment of the 10-13 percent of cystic fibrosis patients who do not respond to currently approved CFTR modulators, as well as RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene. ReCode is expanding its pipeline to develop potential therapies for other rare and common genetic diseases including musculoskeletal, central nervous system, liver and infectious disease indications.

A clinical-stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and address disease drivers at their origin. Remix's REMaster™ technology platform leverages cutting-edge data science, biomolecular sciences and chemistry approaches to identify orally administered compounds that modulate gene expression. Remix's innovative therapeutic approach led to the discovery of REM-422, a first-in-class RNA processing modulator in oncology, now being evaluated in Phase 1 clinical studies to treat acute myeloid leukemia (AML), high-risk myelodysplastic syndrome (HR-MDS) and adenoid cystic carcinoma (ACC). 

Clinical-stage biopharmaceutical company pioneering Regenerative Macrophage Therapy (RMT) in inflammatory and fibrotic diseases. Lead candidate RTX001 is in the clinic for End-Stage-Liver-Disease having demonstrated superior anti-fibrotic and anti-inflammatory efficacy vs non-engineered macrophages in both in vitro and in vivo studies. In addition, the company established two pre-clinical pipeline programs in lung fibrosis and GvHD. Resolution is built on foundational science including two clinical studies from the University of Edinburgh and is led by CEO Dr. Amir Hefni alongside a highly experience transatlantic team. 

Clinical-stage biopharmaceutical company developing next-generation psychedelic-inspired therapeutic solutions for the treatment of major neuropsychiatry indications. Lead candidate, RE104, the only prodrug of 4-OH-DiPT in clinical development, is designed to deliver fast-acting therapeutic benefits with a short duration of psychoactive effects. In August, Reunion announced positive topline results from the RECONNECT Phase 2 trial evaluating RE104 in moderate-to-severe postpartum depression (PPD), demonstrating rapid onset, durable efficacy as an anti-depressant and early signals as an anxiolytic, with a favorable tolerability profile. Based on these data, Reunion plans to advance RE104 into a pivotal Phase 3 trial in PPD in 2026. Reunion is also initiating the REKINDLE Phase 2 trial evaluating RE104 for the treatment of adjustment disorder (AjD) in patients with cancer and other medical illnesses and plans to commence the RECLAIM Phase 2 trial in Generalized Anxiety Disorder (GAD) in the first quarter of 2026. Reunion is also developing RE245, a discovery-stage, non-psychedelic asset, for application in chronic mental health conditions. Reunion recently announced the close of the third and final upsized $51 million tranche for its Series A financing, bringing the total amount raised to $133 million.

Biopharmaceutical company committed to our mission of pioneering solutions to deliver life-changing brain health medicines, so every person can thrive. Sage developed the only two FDA-approved treatments indicated for postpartum depression and is advancing a robust pipeline to target unmet needs in brain health.

Clinical-stage biotechnology company developing AAV-based gene therapies to treat debilitating chronic conditions. Spur’s lead asset, FLT201, is an AAV gene therapy candidate that leverages Spur’s proprietary and potent AAVS3 capsid to deliver GCase85, a rationally engineered longer-acting version of the GCase enzyme missing in Gaucher patients, to stop disease progression and reduce symptoms in Gaucher disease. FLT201 is currently being investigated in a Phase 1/2 trial in Gaucher disease Type 1, with data to date demonstrating favorable safety and tolerability as well as early signals of clinical improvements. The Company expects to initiate a Phase 3 registrational trial in 2025. The Company is also advancing a Phase 1/2 clinical trial for asset SBT101 in adrenomyeloneuropathy (AMN) and preclinical programs in GBA1-linked Parkinson’s disease and severe subset of chronic heart failure.

Clinical-stage company committed to developing new standards of care for the frontline treatment of patients with hematologic malignancies. Driven by the motivation to help patients with blood disorders that have largely eluded other targeted approaches, Syros is developing tamibarotene, an oral selective RARα agonist in frontline patients with higher-risk myelodysplastic syndrome (HR-MDS) with RARA gene overexpression. Ongoing Phase 3 global clinical trial, SELECT-MDS-1, with pivotal data expected by mid-4Q24.

Commercial stage biopharmaceutical company focused on developing therapeutic candidates for highly prevalent diseases with limited treatment options, beginning with eyecare indications. XDEMVY® (lotilaner ophthalmic solution) 0.25% , the first and only U.S. FDA approved prescription treatment for Demodex blepharitis, has continually shown strong quarter-over-quarter sales and revenue growth since launch in August 2023. Well positioned to continue delivering net product sales growth with a projected peak sales potential of >$1B and gross-to-net discounts with robust payer coverage including commercial and Medicare payers, expanded sales force and a planned new direct-to-consumer TV campaign. Advancing clinical development of other pipeline programs and remain on-track to engage with the FDA by year end of 2024 on TP-03, a topical ophthalmic formulation of lotilaner for Meibomian Gland Disease (MGD); TP-04, a topical gel formulation of lotilaner for the treatment of papulopustular rosacea; and TP-05, a lotilaner oral tablet for the prevention of Lyme disease.

Clinical-stage biopharmaceutical company developing a portfolio of orally delivered small-molecule product candidates to address serious diseases. Lead program, TERN-701, an allosteric BCR-ABL inhibitor, is currently in Phase 1 development for potential treatment of chronic myeloid leukemia (CML), with efficacy and safety data including 6-month major molecular response (MMR) rates expected in 4Q25. TERN-601, a small-molecule GLP-1 receptor agonist, for obesity is in Phase 2 development with 12-week efficacy, safety and tolerability data expected in early 4Q25. Additionally, Terns has a Phase 3-ready THR-β agonist, and a preclinical GIPR modulator discovery effort, prioritizing a GIPR antagonist nomination candidate. The company seeks to partner its portfolio of potentially best-in-class metabolic assets while maintaining its focus in oncology and has cash runway into 2028. 

Clinical-stage biopharmaceutical company focused on the discovery and development of transformative medicines for the treatment of serious diseases in areas of high unmet medical need. The Company’s pipeline consists of a family of small molecule compounds called SMTPs (Stachybotrys Microspora Triprenyl Phenols) derived from a fungus. TMS’ lead program, TMS-007 (JX10), has demonstrated efficacy and safety in its Phase 2a study for the treatment of acute ischemic stroke. The Company’s robust pipeline also includes programs in resistant or uncontrolled hypertension, acute kidney injury, and spinal cord injury. TMS continues to explore new pipeline products by leveraging its established partnerships with leading academic institutions in Japan, with the intention to build a bridge between academic discoveries and the global pharmaceutical market.

Clinical-stage cell therapy company leveraging its global presence and core non-viral platform to democratize CAR-T therapy for patients worldwide. Headquartered in San Mateo, California with R&D, manufacturing and clinical operations hubs across Europe and Asia, TriArm is executing on its mission to enable better, safer and more accessible CAR-T products through a four-pronged approach centered around the company’s (1) Fast-in-Time™ (FIT™) non-viral platform capable of CAR-T manufacture in two days, (2) straightforwardly differentiated programs aimed at clinically- and/or commercially-validated targets, (3) rapid and efficient clinical translation and de-risking process via investigator-initiated trials (IITs), and (4) focus on delivering real benefit to CAR-T eligible patients currently marginalized by manufacturability issues and prohibitive cost tied to conventional CAR-T programs.

Clinical-stage biopharmaceutical company engineering conditionally activated cytokines for cancer and other serious diseases. Clinical INDUKINE™ molecules have demonstrated platform proof of concept. WTX-124 (IL-2) shows best-in-class potential in ongoing Ph. 1/1b study: well-tolerated as monotherapy in outpatient setting with clinical activity, with multiple expansion arms now open; strong combination profile observed in dose escalation. WTX-330 (IL-12) induced clinically relevant monotherapy activity at exposures historically toxic; opened expansion arms in advanced or metastatic solid tumors. Updated data from WTX-124 and WTX-330 expected in 4Q 2024. Additional research-stage programs extend platform to a broader range of targets (IL-21, IL-18, IL-10) and indications (inflammation) for future opportunities; cash runway into at least 1Q26.

Clinical-stage biopharmaceutical company advancing a broad a pipeline for cancer and autoimmune diseases, leveraging its XmAb® protein engineering technologies. Wholly owned programs targeting solid tumors include bispecific T cell engagers: ‘819 (ENPP3 x CD3) in ccRCC, ‘808 (B7-H3 x CD28) and ‘541 (CLDN6 x CD3) in ovarian/CLDN6+. Recently added autoimmune pipeline and 4 programs: plamotamab (CD20 x CD3) for RA, XmAb657 (CD19 x CD3), and two targeting TL1A for IBD+. Multiple near-term catalysts, with initiations and clinical readouts expected across the portfolio in 2025. Legacy of partnerships generated >20 XmAb-based candidates in clinical development and 3 marketed products; select partners include Janssen, Amgen, Genentech and more. Company is well-capitalized with runway guided through 2027.

Clinical-stage genetic medicines company focused on redefining the course of neurodegenerative diseases. Lead candidate, PBFT02, is an AAV1 gene therapy that aims to elevate progranulin levels to restore lysosomal function and slow disease progression. PBFT02 is initially targeting genetic forms of frontotemporal dementia (FTD-GRN and FTD-C9orf72) in the ongoing upliFT-D global Phase 1/2 clinical study. Interim data from FTD-GRN patients suggests PBFT02 could be a best-in-class, one-time progranulin-raising therapy. Initial discussions on a registrational study design for FTD-GRN and additional data from the ongoing study are expected in the first half of 2026. The company is also advancing a differentiated preclinical program for Huntington’s disease and has cash runway into 1Q27. 

Emerging biotechnology company pioneering the next generation of RNAi therapies with a unique central nervous system (CNS) pipeline built on a proprietary, three stranded RNA platform named CASi (Conditionally Activated siRNA).  CASi molecules have excellent biodistribution, potency and durability, can be cell-specific and have broad applicability across a range of diseases. Multiple programs are advancing towards the clinic, with one development candidate recently declared, targeting a large market, neurodegenerative indication, with IND filing targeted for next year. A second development candidate will be declared in quick succession in the next quarter. Switch was established on technologies from Caltech, Harvard and City of Hope, launching in March 2023 with $52M, supported by corporate and institutional VCs.

 Focused on the development of emavusertib, an orally available, small molecule, potentially first-in-class IRAK4 inhibitor, in lymphoma, leukemia and solid tumors. Clinical development ongoing in Phase 1/2 TakeAim Lymphoma trial in R/R PCNSL in combination with ibrutinib, Phase 1/2 TakeAim Leukemia trial as a monotherapy in R/R AML and R/R hrMDS, and also as frontline combination therapy with venetoclax and azacitidine in AML. FDA has granted orphan drug designation in PCNSL, AML and MDS. Expanding into CLL, with the potential to enable complete remission or minimal residual disease negativity and time limited treatment, which would be a paradigm shift.  Plans to initiate a combination study of emavusertib plus a BTK inhibitor in CLL by YE25, with initial data expected mid-2026. Updated data from PCNSL expected in 4Q25 to support accelerated filings in both the US and EU. 

Clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for patients with cancer and autoimmune diseases, has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Company’s immuno-oncology and autoimmune pipeline includes iPSC-derived natural killer (NK) cell and T-cell product candidates, which are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple therapeutic mechanisms to patients.

Clinical-stage biopharmaceutical company pioneering the discovery and development of a new generation of immuno-oncology therapeutics. Phase 3 trial underway for belrestotug, a highly potent, FcγR-engaging anti-TIGIT antibody, in collaboration with GSK for NSCLC, with additional late-stage trials ongoing including a Phase 2 trial in HNSCC. Wholly owned program inupadenant, an adenosine A2A receptor antagonist, is currently in a randomized Phase 2 trial in NSCLC patients in combination with chemotherapy. EOS-984, a first-in-class small molecule targeting a novel mechanism in the adenosine pathway, is currently treating patients with advanced solid tumors in a Phase 1 trial. Strong cash balance of ~$714M as of June 30, 2024, expected to provide runway into 2027.

Clinical-stage allogeneic cell therapy and genetic medicines company advancing differentiated non-viral treatments for patients with cancer and rare diseases. Validating big pharma relationships, including cell therapy-focused strategic collaborations with Roche and Astellas. Three clinical stage allogeneic CAR-T programs include Roche-partnered P-BCMA-ALLO1 for multiple myeloma and a dual CAR program, P-CD19CD20-ALLO1, for B-cell malignancies and wholly owned P-MUC1C-ALLO1 in solid tumors. Interim clinical data for P-BCMA-ALLO1 presented at IMS in September 2024 demonstrated high response rates, was well tolerated and available on demand, supporting recently initiated Phase 1b trial and the Company's belief that T stem cell (TSCM)-rich allogeneic CAR-Ts have the potential to address unmet needs in multiple myeloma safely, effectively and reliably. P-BCMA-ALLO1 has received ODD for MM and RMAT designation for adult patients with RRMM from FDA. Also advancing multiple preclinical non-viral genetic medicines programs, including P-FVIII-101 for Hemophilia A and P-KLKB1-101 for hereditary angioedema (HAE). Data updates across pipeline anticipated in Q4 2024.

Leading biotechnology company dedicated to creating and delivering the next-generation of gene editing therapies to patients. Prime Medicines’ proprietary Prime Editing technology enables precise, versatile, and efficient correction of disease-causing mutations, while minimizing unwanted DNA modifications. Currently, Prime is advancing two lead in vivo programs to cure two of the largest genetic liver diseases, Wilson’s Disease and Alpha-1 Antitrypsin Deficiency (AATD), with initial clinical data from both programs expected in 2027. Additionally, Prime continues to advance its Cystic Fibrosis (CF) program with support from the Cystic Fibrosis Foundation, and efforts to develop Prime Edited CAR-T products for hematology, immunology and oncology in partnership with Bristol Myers Squibb. In August 2025, closed a $144.2M follow-on financing, extending cash runway into 2027.

 Akero Therapeutics is a clinical-stage company developing transformational treatments for patients with serious metabolic diseases marked by high unmet medical need, including MASH. Akero’s lead candidate, efruxifermin (EFX), is currently being evaluated in three ongoing Phase 3 clinical studies: SYNCHRONY Histology in patients with pre-cirrhotic (F2-F3 fibrosis) MASH, SYNCHRONY Outcomes in patients with compensated cirrhosis (F4) due to MASH, and SYNCHRONY Real-World in patients with MASH or MASLD (metabolic dysfunction associated steatotic liver disease). The Phase 3 SYNCHRONY program builds on the results of two Phase 2b clinical trials, the HARMONY study in patients with pre-cirrhotic MASH and the SYMMETRY study in patients with compensated cirrhosis due to MASH.  

Clinical-stage biotechnology company committed to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Tenaya’s pipeline includes clinical-stage candidates TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), currently in Phase 1b/2 development, and TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), in Phase 1b development. Data readouts from both TN-201 and TN-401 clinical programs are expected in 4Q25. Tenaya has employed a suite of integrated internal capabilities, including modality agnostic target validation, capsid engineering and manufacturing, to generate a portfolio of novel medicines based on genetic insights, including TN-301, a clinical-stage small molecule HDAC6 inhibitor for the potential treatment of heart failure and related cardio/muscular disease, and multiple early-stage programs in preclinical development aimed at the treatment of both rare genetic disorders and more prevalent heart conditions. 

Late-stage biotech focused on developing best-in-class therapies for neurological and psychiatric disorders. The Company’s late-stage program, valiloxybate, is positioned to become the only no-sodium, once-nightly oxybate therapy for the treatment of narcolepsy and idiopathic hypersomnia, addressing key unmet needs in a $2B market despite limitations of current therapies. XWPharma has aligned with the FDA in an End-of-Phase 2 on the protocol for a single pivotal Phase 3 trial for narcolepsy and is preparing to initiate the study. The Company’s second clinical program, XW10508, is an oral NMDA antagonist for treatment-resistant depression with a differentiated adverse event profile compared to the approved esketamine intranasal therapy, which is anticipated to be a global blockbuster despite its restrictive REMS requiring clinic-based administration. XW10508 has demonstrated safety and tolerability in Phase 1, and an exploratory Phase 2 trial is underway to assess efficacy, safety, and dosage ahead of a subsequent double-blind Phase 2 in the U.S.

Late-stage clinical biotechnology company focused on the discovery and development of drugs for the treatment of adult and pediatric cancer. The company is utilizing its novel proprietary Phospholipid Drug Conjugate™ (PDC) delivery platform to develop the next-generation of cancer cell-targeting treatments, delivering enhanced efficacy with fewer off-target effects resulting in improved safety. The company’s lead asset, iopofosine I 131, is a small-molecule PDC radiotherapeutic designed to provide targeted delivery of the radioisotope iodine-131. Iopofosine I 131 has demonstrated clinical activity in hematologic malignancies as well as tumors located across the blood brain barrier. Recently, the pivotal study in Waldenstrom’s macroglobulinemia (WM) was successfully completed and the NDA is being compiled. The company anticipates receiving an accelerated review which, assuming marketing approval, supports a projected commercial launch in the second half of 2025. WM represents a meaningful market and the company plans to expand iopofosine I 131 into additional liquid tumors while selectively advancing its robust pipeline of radiopharmaceuticals in liquid and solid tumors.

Pivotal-stage biotechnology company focused on developing pozdeutinurad (AR882), a potentially best-in-class, highly potent and selective next-generation URAT1 inhibitor to reduce serum urate levels, flares, and tophi in patients with gout. Gout remains a large and growing market with ~ 13M patients in the U.S. alone, ~2M of which have tophaceous gout.  Significant commercial opportunity with potential to achieve >$2.0B in peak revenue. Pozdeutinurad has demonstrated encouraging efficacy and safety compared to SOC in Phase 2 studies. A Phase2b study to evaluate pozdeutinurad in tophaceous gout patients showed impressive results, with 29% of patients receiving pozdeutinurad monotherapy having a complete resolution of target tophi. Granted Fast Track Designation in September 2024 and completed enrollment in both Phase 3 programs, REDUCE 1 and REDUCE 2, ahead of schedule and plan to share pivotal data Q2 2026.

CVAC is a multinational biopharmaceutical company pioneering transformative mRNA-based medicines to address areas of high unmet medical need, with a primary focus on oncology and infectious diseases. Oncology pipeline features CVGBM, an off-the-shelf shared-antigen cancer vaccine candidate targeting glioblastoma, with promising Phase 1 data presented at ESMO/SITC 2024 and ongoing dose-expansion enrollment. By late 2025, CureVac expects to bring a second shared-antigen candidate in squamous NSCLC into the clinic. In infectious diseases, CureVac recently initiated a program for urinary tract infections, one of the most prevalent bacterial infections globally, based on promising preclinical results. Its licensed prophylactic vaccine portfolio with GSK includes a seasonal flu mRNA vaccine demonstrating positive Phase 2 data in Q3 2024, set to enter Phase 3 in 2025, a Phase 2 avian influenza (H5N1) study, granted FDA Fast Track Designation with data expected in H1 2025, and a newly initiated flu/COVID-19 combo Phase 1/2 study. CureVac’s innovative mRNA platform, broad R&D capabilities, strategic partnerships, and financial stability, supported by a recent €400 million payment from GSK, underline its potential to deliver next-generation therapies and vaccines​.

Clinical-stage biotechnology company developing novel radiopharmaceutical therapies against cancer. The lead product candidate, Radspherin, uses the alpha-emitting radionuclide radium-224, directly targeting micro-metastases post-surgery, harnessing the benefits of modern radiopharmaceuticals without the complexities of biological targeting. Oncoinvent is investigating the safety and efficacy of Radspherin in a clinical development program in two indications.  In addition to the finalized Phase 1/2a trial in colorectal cancer, one Phase 1 trial and one randomized Phase 2 trial, both in ovarian cancer, are ongoing in the US, UK and Europe. Preliminary clinical efficacy data are highly encouraging, and no serious toxicity or safety concerns have been reported to date.

SURGE Therapeutics (Private) is a clinical-stage biotechnology company pioneering intraoperative immunotherapy with its proprietary SURGERx platform. Their approach focuses immunotherapy where and when it should work best – namely, at the site and time of tumor resection – to prevent post-surgical recurrence and metastasis. Reimagining surgical oncology from being a physical intervention only into a therapeutic intervention as well represents an enormous market opportunity. SURGE has multiple Phase 1 clinical programs underway, assessing two distinct clinically validated APIs across three indications, with Phase 2 studies on track to be initiated in 2026. 

Radiopharmaceutical company providing rare medical radioisotopes, patient dose manufacturing and development services to the Radiopharmaceutical market. Radiopharmaceuticals deliver precision diagnosis and therapy to treat serious disease such as cancer.  NorthStar is emerging as the largest commercial scale manufacturer of Copper-67 and non-carrier added Actinium-225.  We will provide patient dose manufacturing services from our 53,000 square foot CDMO facility.  In addition, NorthStar is providing value adding research and development services for our customers.  NorthStar performs this work in state of the art, built for purpose facilities.  All of our products and services are offered in a unique, one-campus concept.  NorthStar has established a leading book of business through long-term contracts with our biotech and big pharma customers.  NorthStar has built an outstanding team of 250 + individuals who daily contribute to our mission of providing patients global access to game changing radiopharmaceuticals.

Biotechnology company with operations in Europe and the U.S. that is dedicated to transforming patient outcomes through life-changing science and innovation. Focusing on high unmet medical needs, Galapagos synergizes compelling science, technology, and collaborative approaches to create a deep pipeline of best-in-class small molecules and cell therapies in oncology and immunology. With capabilities from lab to patient, including a decentralized cell therapy manufacturing platform, and the financial strength (>€3B) to invest strategically for the near- and long-term, Galapagos is committed to challenging the status quo and delivering results for patients, employees, and shareholders.

TCR-based drug development company with two near term clinical stage assets in acute myeloid leukemia (AML).  BlueSphere is leveraging its proprietary TCR discovery platform, TCXpress™, to build both its own pipeline and strategic partnership opportunities. The initial clinical candidate, BSB-1001 (from the TCX-101 program), targets the minor histocompatibility antigen-1 (HA-1), and will be dosed with allogeneic hematopoietic stem cell transplant.  The company anticipates enrolling the first patient in 1Q25.  A second clinical candidate, BSB-2001 (from the TCX-102 program) targets AML patients with mutant NPM1, will be autologous and not given in combination with stem cell transplant.  An IND is expected in the second quarter of 2025.

Biopharmaceutical company harnessing next-generation engineering of small interfering RNAs (siRNAs) to improve and expand the reach of RNAi-based medicines. The Company is building a pipeline of innovative RNAi therapeutics to make a significant impact for patients across multiple therapeutic areas. Co-founded by pioneering executives and scientists in RNAi, including executive chair John Maraganore. The Company has research collaborations in place with Biogen and Bausch + Lomb and is backed by leading life sciences investors, including ARCH Venture Partners, Fidelity Management & Research Company, Invus, Slate Path Capital, and Rock Springs Capital.

Clinical-stage oncology company dedicated to redefining the future of care through targeted radiotherapeutics for patients with aggressive and hard-to-treat cancers. The company’s lead program, 225Ac-SSO110, a novel antagonist of the somatostatin type 2 receptor (SSTR2) with best-in-class potential, is currently being investigated in the Phase 1/2 SANTANA-225 study as the first maintenance radiotherapy for extensive stage small cell lung cancer (ES-SCLC) and Merkel Cell Carcinoma (MCC) –two diseases with limited options and poor prognosis. Ariceum is also developing ATT001, a novel radiolabeled I-123 PARP inhibitor designed to deliver subcellular precision radiotherapy to aggressive solid tumors. Headquartered in Berlin, Ariceum operates across Germany, Switzerland, Australia, the United Kingdom, and the United States. The company is supported by leading global life sciences investors, including EQT Life Sciences, HealthCap, Pureos Bioventures, Andera Partners, and Earlybird Venture Capital.

Biotechnology company pioneering an entirely new dual agonistic, multifunctional and avidity engineered antibody modality, “AVC-Boosters,” designed to safely deliver targeted and potent immune-modulating monotherapy for cancer patients. The AVC-Booster design surpasses the best qualities of first-generation antibodies, checkpoint inhibitors, T-cell engagers and antibody-drug conjugates (ADCs) to unlock strong and orchestrated immunological responses, harnessing the full power of the innate and adaptive immune system. The company’s lead oncology program, AVC-S-101, is a TROP2-targeting booster for the treatment of non-small cell lung cancer and several other cancer indications. Avidicure is based the Netherlands and supported by a syndicate of top-tier investors led by EQT-LS. 

Clinical stage medical device company focused on the development of novel, minimally invasive treatments for patients with neurological diseases. CereVasc's initial product, the eShunt® System, encompasses groundbreaking percutaneous transvenous-transdural access to the central nervous system intended to allow for minimally invasive treatment for communicating hydrocephalus (CH). As demonstrated in pilot studies, this is a promising improvement to the 60+-year-old standard of care which involves open brain surgery. CereVasc is currently evaluating the eShunt System in a pivotal trial for the treatment of elderly patients with normal pressure hydrocephalus (NPH), a condition that exerts pressure on the brain due to an accumulation of cerebrospinal fluid (CSF), leading to side effect such as cognitive dysfunction and gait disturbance. 

Merida is advancing a precision immunology approach for antibody-driven diseases that selectively and durably eliminate their pathogenic drivers. This approach, enabled by advances in protein engineering and immunology, has the potential to address numerous autoimmune and allergic conditions that are inadequately treated today. The Company is pursuing development in Graves’ Disease, IgE-mediated allergic diseases, and primary membranous nephropathy. Merida launched in April 2025 with a $121M Series A co-led by Bain Capital Life Sciences, BVF Partners and Third Rock Ventures, joined by GV and Perceptive Xontogeny Venture Funds.

A clinical-stage biotechnology company developing one-time in vivo genome-editing therapies for serious genetic, metabolic, infectious, and autoimmune diseases. Its proprietary platform—comprising CRISPR nucleases (YolCas), base editors (YolBE), and lipid nanoparticle delivery (Yol-LNPs)—together with a cGMP-compliant manufacturing facility supporting clinical supply through Phase III and early commercial scale-up, underpins one of the most extensive in vivo gene-editing clinical pipelines globally.
YolTech's clinical-stage portfolio includes lead asset YOLT-201 for transthyretin amyloidosis (ATTR), YOLT-101 for familial hypercholesterolemia (HeFH), YOLT-203 for primary hyperoxaluria type 1 (PH1), YOLT-204 for transfusion-dependent β-thalassemia and sickle cell disease (TDT/SCD), YOLT-202 for alpha-1 antitrypsin deficiency (AATD) and YOLT-401, an in vivo CAR-T program approaching clinical stage for autoimmune diseases

 Clinical-stage biotechnology company focused on addressing unmet needs in cancer through metabolism, and founded by world-leading scientists, including Lew Cantley, discoverer of the PI3K pathway. Lead clinical program aims to disrupt the PI3K/AKT/mTOR pathway - the most frequently mutated in cancer – with an oral, multi-node approach designed to widen the therapeutic window and improve outcomes. Phase 1b data of PIKTOR in combination with paclitaxel in patients with PI3K-mutated solid tumors demonstrated 47% ORR, including 3 CRs, with 80% ORR in endometrial cancer, and PFS of 11 months. Initiated a Phase 2 trial in 2L R/R endometrial cancer based on these results, with data expected in early and mid-2026. Competitively positioned following recent positive clinical readouts in PI3K wild type patients. Backed by leading investors, including Khosla Ventures, Future Ventures and Digitalis Ventures. 

First dedicated ophthalmology hub-and-spoke company developing a de-risked, balanced portfolio of late preclinical and clinical-stage assets targeting both front and back-of-the-eye diseases. Advancing a diversified pipeline of therapies and devices through focused subsidiaries, to reduce risk, improve efficiency and scale impact. Founded by a team with 100+ combined years of ophthalmology experience and 30+ successful launches and exits. Debuted at the Ophthalmology Futures European Forum in 2025 and completed seed financing led by ClavystBio.

Clinical-stage biotechnology company developing pipeline of potentially best-in-class therapies to transform the standard of care for patients with autoimmune diseases. Lead candidate, JADE101, an anti A PRoliferation-Inducing Ligand (APRIL) monoclonal antibody, is being evaluated for the treatment of IgA nephropathy in a Phase 1 clinical trial. In September 2025, Jade announced the first cohort dosed in healthy volunteers and expects interim, biomarker rich data in 1H26.  In October 2025, Jade unveiled JADE201, a half-life extended, afucosylated anti-BAFF-R antibody designed for durable B-cell depletion and broad autoimmune potential, with a first-in-human trial in rheumatoid arthritis planned for 1H26. Pipeline also includes JADE-003, an undisclosed antibody program targeting clinical trial initiation in 1H27. In April 2025, Jade went public via a reverse merger with Aerovate Therapeutics and raised approximately $300M through an oversubscribed private investment round. Following a recent additional private placement of $135M, the company has cash runway into 1H28.

Developing and commercializing life-changing therapies for people living with gastrointestinal and rare diseases. Pioneer in the development of LINZESS® (linaclotide), the U.S. branded prescription market leader for patients with IBS-C or CIC, which continues to deliver meaningful prescription demand growth. Advancing apraglutide, a next-generation, long-acting synthetic GLP-2 analog being developed for short bowel syndrome patients who are dependent on parenteral support. 

Stealth Biotherapeutics' mission is to develop novel therapies to improve the lives of patients living with diseases of mitochondrial dysfunction. Stealth’s commercial product, FORZINITY, was granted accelerated approval by the U.S. Food & Drug Administration (FDA) in September 2025 as the first FDA-approved treatment for Barth syndrome, as well as the first FDA-approved mitochondria-targeted therapeutic. In connection with the approval, Stealth was awarded a Rare Pediatric Disease Priority Review Voucher. Stealth is studying FORZINITY (elamipretide) in additional indications, including dry age-related macular degeneration and primary mitochondrial myopathy, and is developing its second-generation clinical-stage candidate, bevemipretide (SBT-272), an eye drop formulation for ophthalmic and neurological disease indications.

Leading clinical-stage gene editing company leveraging CRISPR-based technology to develop novel, one-time therapies to treat patients at root cause of their disease and deliver superior outcomes. Three Phase 3 trials are ongoing across two programs: Lonvo-z in HAE, for which enrollment in HAELO is complete, topline data is expected in 1H26 and BLA is expected in 2H26; and two Phase 3s of Nex-z for ATTR, with enrollment completion in MAGNITUDE-2 (PN) expected in 1H26 and MAGNITUDE (CM) by early 2027. Long-term data from earlier studies have demonstrated potential to address significant unmet need and transform chronic disease management; additional data from both programs are expected in 4Q25. Intellia is actively building the infrastructure to support its first planned commercial launch in 2027.

Walden Biosciences (Private) is developing novel, disease-modifying medicines to treat kidney diseases that apply novel, multi-disciplinary approaches, which directly target the kidney to prevent damage, slow disease progression, and restore kidney function. Lead product, WAL0921, is a humanized monoclonal antibody that inhibits suPAR, a cause of podocyte dysfunction and kidney disease, that has demonstrated proof-of-concept in an ongoing Phase 2 Basket Study for the treatment of rare chronic kidney diseases.  Early data readout from the rare cohorts of the Phase 2 Basket study are expected in 4Q25.  WAL0623 is an IND-ready small molecule designed to stabilize and restore the function of dynamin, an enzyme responsible for the maintenance of the cytoskeletal architecture and function of podocytes and proximal tubule cells.  Walden’s product candidates are well-differentiated from current therapies as they are not hemodynamic modulators and are non-immunosuppressive. Both of Walden’s programs offer the promise to deliver disease-modifying, breakthrough therapies that are readily combinable with the standard of care to transform the treatment of kidney disease. 

Pre-NDA stage biopharma company aiming to improve surgical outcomes. Through locally administered, controlled, prolonged-release therapeutics, PolyPid's proprietary PLEX (Polymer-Lipid Encapsulation matriX) technology pairs with Active Pharmaceutical Ingredients (APIs), enabling precise delivery of drugs at optimal release rates over durations ranging from several days to months. In a recent pivotal Phase 3 trial PolyPid’s lead product, D-PLEX₁₀₀, tested for the prevention of abdominal colorectal surgical site infections, achieved a 38% reduction (p<0.005) in the combined endpoint of surgical site infections, reinterventions, or mortality compared to standard of care alone. Additionally, the trial showed a 58% reduction (p<0.005) in the rate of surgical site infections in the D-PLEX₁₀₀ treatment arm. New Drug Application (NDA) submission is expected in early 2026. In addition, the Company has an innovative pipeline in oncology, obesity, and diabetes. 

Late-stage biopharma company developing emactuzumab, a next-generation monoclonal antibody targeting CSF-1R for TGCT and other macrophage-driven diseases. Emactuzumab is currently being evaluated in the global Phase 3 TANGENT trial for TGCT patients with top-line data expected in Q1 2026. Clinical data to date supports its potential for a short treatment cycle, rapid onset, and durable response—differentiating it from chronically administered oral agents. SynOx operates across Dublin, Oxford, and Philadelphia, and is backed by leading life science investors including Forbion, Gilde Healthcare, HealthCap, Bioqube Ventures, and Medicxi.

Clinical-stage immuno-oncology company focused on activating the innate immune system in the fight against cancer. Founded on breakthrough research from Dr. Irv Weissman’s lab at Stanford University and led by scientific experts in immunology and cancer immunotherapy, Pheast is developing novel therapies for some of the most difficult-to-treat and aggressive cancers. Pheast’s lead program, PHST001, targets a novel immune checkpoint pioneered by the Pheast founders. It is currently in a Phase 1 clinical trial for the treatment of advanced solid tumors, with planned expansion into various CD24+ cancers in 1H-2026. Beyond its lead program, Pheast is developing a broad oncology pipeline that includes highly differentiated bispecific ADCs. The company is backed by leading life sciences investors, including Catalio Capital Management and ARCH Venture Partners.

Commercial-stage medical dermatology company addressing the urgent needs of individuals living with immune-mediated dermatological diseases. Commercially marketed asset, ZORYVE®, is approved in plaque psoriasis, atopic dermatitis, and seborrheic dermatitis with multiple formulations and dosages. The company’s strategy is focused on driving sustained growth for ZORYVE in currently approved indications, expanding into new disease areas with ZORYVE, and building its innovative pipeline including advancing ARQ-234, a differentiated biologic for atopic dermatitis. 

Private, oncology-focused biotherapeutics company developing genetic medicines to transform the treatment of solid tumors. CorriXR’s proprietary in vivo gene editing tool disables the transcription factor NRF2, disrupting cancer cell survival mechanisms and sensitizing them to standard of care (SOC) treatments. Preclinical proof of concept (POC) for the platform has been successfully demonstrated, and IND-enabling work is ongoing in CorriXR’s lead program CXR101 for the treatment of head & neck squamous cell carcinoma. CXR101 is designed to seamlessly integrate with current SOC, either as a neoadjuvant therapy to shrink tumors ahead of surgery, or in combination with chemo, radiotherapy or immunotherapy treatment, where it has the potential to improve treatment effectiveness and efficiency at lower doses. CorriXR is the first biotech spinout from ChristianaCare’s Gene Editing Institute (GEI), and has a highly experienced, lean management team and strong financial position, with continued clinical backing and operational support from ChristianaCare.