JP Morgan 2027

Clinical-stage biotechnology company advancing two differentiated oncology programs, evorpacept, a novel CD47-inhibitor designed to enhance the activity of anti-cancer antibodies while mitigating hematologic toxicity and safety limitations observed with first-generation CD47 agents, and ALX2004, the only matuzumab-based EGFR antibody-drug conjugate (ADC) in development. Evorpacept is being evaluated in combination with trastuzumab and chemotherapy in a biomarker-driven Phase 2 trial in patients with metastatic HER2-positive breast cancer, with topline data from the first 80 patients expected in mid-2027. ALX2004 is engineered to enhance tumor delivery while minimizing off-tumor toxicity in EGFR-expressing solid tumors, with Phase 1 initial safety data expected in the second half of 2026. ALX retains worldwide rights to both programs and has projected cash runway through the first half of 2028.

Clinical-stage biopharmaceutical company developing inhaled, lung-targeted therapies for pulmonary fibrosis and other serious, rare respiratory diseases. Advancing a pipeline of optimized inhaled antifibrotic medicines designed to improve tolerability and reduce systemic side effects, including AP01 (inhaled pirfenidone), AP02 (inhaled nintedanib), and AP03, an inhaled fixed-dose combination therapy. Topline data from ongoing trials of AP01 and AP02 are anticipated in 2H and late 2027, respectively. Leveraging its proprietary drug-device platform and expertise in rare respiratory disease development, Avalyn is working to redefine the standard of care for pulmonary fibrosis through direct-to-lung delivery of established antifibrotic medicines.

Avere Therapeutics is a biotechnology company developing oral therapies for the treatment of IL-23- driven inflammatory diseases. Avere’s lead asset, AVR-001, is an oral IL-23 receptor antagonist with the potential to deliver competitive efficacy with emerging oral IL-23 therapies in a convenient, once-weekly dose. Avere is initially advancing AVR-001 in psoriasis, with potential to expand into multiple indications, including ulcerative colitis, Crohn’s disease, and psoriatic arthritis, where the IL-23 pathway is validated and the need for more convenient and effective therapies remains high.

Clinical-stage biopharmaceutical company focused on transforming care for the treatment of inflammatory diseases. Lead candidate, rademikibart, is a potentially best-in-class, next-generation anti-IL-4Rα antibody being developed for the treatment of acute exacerbations of asthma and COPD, with potential to expand into chronic indications over time. Reported positive preliminary topline results from Phase 2 Seabreeze STAT asthma and COPD studies in September 2026. In asthma, rademikibart meaningfully reduced treatment failure at 28 days by 66% compared to placebo and demonstrated a statistically significant rapid improvement in post-bronchodilator FEV1 at Day 7 compared to placebo. In COPD, rademikibart significantly reduced treatment failure by 81% through week 4, significantly reduced the rate of new moderate to severe COPD exacerbations through one month by 85%, and significantly reduced the rate of hospitalizations or ED visits for new acute exacerbations by 100% compared to placebo. Rademikibart was well tolerated in both trials. Plan to engage with U.S. FDA to gain alignment on a Phase 3 development pathway in both indications.

Clinical-stage biopharmaceutical company building a portfolio of innovative, potentially 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, targeting the underlying cause of disease for erythropoietic protoporphyria (EPP). Disc has additional pipeline candidates focused on iron modulation – including DISC-0974, an anti-hemojuvelin (HJV) antibody designed to suppress production of hepcidin and enhance iron levels, targeting anemias in myelofibrosis (MF) and anemia of inflammatory bowel disease (IBD), as well as DISC-3405 targeting Transmembrane Serine Protease 6 (TMPRSS6) designed to induce hepcidin expression for diseases enabled by iron availability, including polycythemia vera (PV) and sickle cell disease (SCD).

Late-stage clinical biopharmaceutical company building a global, fully integrated organization focused on developing innovative medicines for cancer and other serious unmet needs. Pipeline includes differentiated PARP1-selective franchise comprised of EIK1003 and CNS-penetrant EIK1004 to expand beyond current maintenance treatment, EIK1001, a systemically administered dual TLR7/8 agonist advancing toward registrational development in melanoma and NSCLC, designed to complement checkpoint inhibition, and EIK1005, a WRN inhibitor being developed for MSI-high tumors, in Phase 1/2. Leveraging deep R&D expertise and a proprietary single-molecule tracking platform, Eikon is pursuing previously undruggable targets and uncovering novel biology.

Biotechnology company pioneering targeted biologics-based conditioning regimens to make hematopoietic stem cell transplantation (HSCT) safer, more tolerable, and widely accessible. Inograft is developing a purpose-built platform designed to unlock the full potential of HSCT by overcoming the key barriers that limit its use, starting with conditioning. Inograft was founded by trailblazers in the field of HSCT, including Dr. Irv Weissman, and builds on 30+ years of foundational research at Stanford. The company is backed by leading life sciences investors including ARCH Venture Partners, Sutter Hill Ventures, and Frazier Healthcare Partners, and is advancing its lead conditioning program into clinical development in 2027.

Biotechnology company focused on developing and commercializing therapies for gastrointestinal and rare diseases. Commercial product, LINZESS® (linaclotide), is the leading branded prescription therapy in the U.S. for adults with irritable bowel syndrome with constipation (IBS-C) expected to generate $1.15-1.2B in 2026 US net sales. Apraglutide, Ironwood’s long-acting, once-weekly GLP-2 analog, has potential to be a best-in-class treatment for short bowel syndrome with intestinal failure (SBS-IF). The confirmatory Ph 3 STARS-2 trial of apraglutide in adults with SBS-IF is recruiting patients to support a 2029 NDA submission. STARS-2 builds on positive results from the Phase 3 STARS study and the STARS-Extend long-term extension study. 

Clinical-stage biotechnology company developing pipeline of potentially best-in-class therapies that address critical unmet needs for patients with autoimmune diseases. Lead candidate, JADE101, an anti-APRIL monoclonal antibody for IgA nephropathy (IgAN), reported positive interim Phase 1 data supporting every-12-week subcutaneous dosing and best-in-class activity. Phase 2 trial of JADE101 is underway, with interim data expected in 2027 and a registrational Phase 3 planned for 1H27. Pipeline also includes JADE201, an afucosylated anti-BAFF-R monoclonal antibody, in development for multiple systemic autoimmune diseases; and JADE301, an anti-IFN-β monoclonal antibody being evaluated for the treatment of dermatomyotis. Jade was launched based on assets licensed from Paragon Therapeutics, an antibody discovery engine founded by Fairmount.

Clinical-stage biopharmaceutical company dedicated to the development of novel therapeutics for diseases with high unmet medical needs, including treatment-resistant metastatic breast cancer and amyotrophic lateral sclerosis (ALS), with the goal of improving patients’ lives. The Company's lead drug candidates, lasofoxifene and brelgometon (ATH-1105), are novel, small molecule therapies with the potential to address devastating diseases where current treatment options are limited or ineffective. Lasofoxifene is a novel, nonsteroidal selective estrogen receptor modulator (SERM) with a unique binding profile, designed to provide potent activity against both wild-type and mutant estrogen receptors, including the clinically significant ESR1 mutations commonly associated with resistance to endocrine therapy in metastatic breast cancer. Lasofoxifene is being advanced in a Phase 3 clinical trial as a targeted therapy for ER+ HER2-negative, ESR1-mutated metastatic breast cancer, which is expected to complete enrollment in the fourth quarter of 2026 and to have topline data in 2027.

Mighty Therapeutics (f/k/a Stealth BioTherapeutics) is advancing novel therapies for people living with diseases involving mitochondrial dysfunction. Grounded in rigorous science and inspired by meaningful patient partnerships, the company is building a proprietary pipeline to directly address bioenergetic deficits at the source. In September 2025, Mighty received U.S. Food and Drug Administration (FDA) approval of its first commercial therapy, establishing both the first FDA-approved treatment for Barth syndrome and the first FDA-approved therapy to directly target mitochondria. Mighty continues to develop elamipretide in Barth syndrome, polymerase gamma (POLG) related mitochondrial disease, and dry AMD. Mighty is also progressing its next-generation clinical candidate, bevemipretide, for ophthalmic and neurological pathologies, and continues to develop preclinical assets SBT-255 and SBT-589 for rare mitochondrial disorders.

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. Neumora is currently advancing three programs that target novel mechanisms of action for a broad range of underserved, prevalent diseases; NMRA-511 (V1aR Antagonist) in Alzheimer’s disease (AD) agitation with clinical data expected in 4Q26, NMRA-898 (M4 Positive Allosteric Modulator) in schizophrenia with clinical data expected in 2H26, and NMRA-215 (NLRP3 Inhibitor) for obesity with plans to submit IND in 4Q26 and initiate a Phase 1 study by the end of 2026.

NewAmsterdam Pharma (NAMS) is a late-stage biopharmaceutical company whose mission is to improve patient care in populations with metabolic diseases where currently approved therapies have not been adequate or well tolerated. In multiple phase 3 studies, NewAmsterdam is investigating obicetrapib, an oral, low-dose and once-daily CETP inhibitor, alone or as a fixed-dose combination with ezetimibe, as preferred LDL-C lowering therapies to be used as an adjunct to statin therapy for patients at risk of CVD with elevated LDL-C, for whom existing therapies are not sufficiently effective or well tolerated.

Leading biotechnology company dedicated to creating and delivering the next-generation of gene editing therapies. Advancing lead in vivo programs to cure two of the largest genetic liver diseases, Wilson Disease and Alpha-1 Antitrypsin Deficiency (AATD), with initial clinical data from both programs expected in 2027. Additionally, following positive proof-of-concept data in CGD, expect to file a BLA for PM359 in 1H 2027. Continuing to advance 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; over time, intend to maximize Prime Editing’s broad and versatile therapeutic potential to rapidly and efficiently expand beyond current pipeline.

Clinical-stage biopharmaceutical company advancing a novel pipeline of first-in-class, multi-pathway therapies to address the underlying drivers of disease in orphan pulmonary and fibrosis indications. Lead program, LTI-03, is synthetic peptide that mimics Caveolin-1, a key regulator of multiple fibrosis-related pathways. LTI-03 has blockbuster potential and is designed with a unique dual mechanism of action targeting both alveolar epithelial cell regeneration and inhibition of profibrotic signaling, addressing key drivers of disease progression in Idiopathic Pulmonary Fibrosis (IPF). Approved therapies only slow disease but do not prevent or reverse fibrosis progression and there remains significant unmet need within the >$11B global IPF market. Phase 1 clinical data demonstrated a favorable safety profile, dose flexibility, and validated biomarker activity linked to lung function in IPF. LTI-03 is actively enrolling patients in the global Phase 2 RENEW trial in IPF, and interim data is expected in 2H 2026 with topline data expected in 2027.

Clinical-stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and address disease drivers at their origin. Lead asset, REM-422, is a first-in-class orally available mRNA degrader of the oncogenic transcription factor MYB, now being evaluated in clinical studies in both Adenoid Cystic Carcinoma (ACC) and Acute Myeloid Leukemia (AML) or high-risk myelodysplastic syndrome (HR-MDS). Positive Phase 1/2 results for REM-422 in ACC showed a 43% overall response rate in biomarker-positive patients, 100% disease control, and responses lasting more than one year with treatment ongoing. Remix is conducting an ongoing registrational directed study in ACC with data expected in 2027. In June 2026, Remix announced a merger with Passage Bio (PASG), which is expected to close in the coming weeks. 

Clinical-stage biopharmaceutical company focused on developing innovative therapies for underserved mental health disorders. Lead program, luvesilocin (RE104), is a proprietary, patented, short-duration prodrug of 4-OH-DiPT, a 5-HT2A receptor agonist, purpose-designed to strike the optimal balance between therapeutic intensity, patient experience, and clinical practicality. The company is advancing RE104 through a deliberate clinical development strategy designed to generate insights across mood and anxiety disorders, inform future indications, and maximize long-term patient impact. Reunion is currently evaluating RE104 in three lead indications: Generalized Anxiety Disorder (GAD) through the ongoing Phase 2 RECLAIM trial, Adjustment Disorder (AjD) through the ongoing Phase 2 REKINDLE trial, and Major Depressive Disorder (MDD) & Postpartum Depression (PPD), with a Phase 3 study expected to initiate.

Clinical-stage biotechnology company committed to a bold mission: 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); TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC); and TN-301, a highly specific small molecule HDAC6 inhibitor with broad potential clinical utility in cardiac, metabolic and muscular conditions, including heart failure with preserved ejection fraction (HFpEF) and Duchenne muscular dystrophy (DMD). Tenaya has employed a suite of integrated internal capabilities including modality agnostic target discovery and validation, to generate a portfolio of novel medicines based on genetic insights, aimed at the treatment of both rare genetic disorders and more prevalent heart conditions.

Tenvie Therapeutics is a clinical-stage biotechnology company developing precision-engineered small molecule therapies for neurological and peripheral diseases driven by neuroinflammation and cellular dysfunction. Using its proprietary product engine, Tenvie is advancing a broad pipeline of CNS-penetrant and peripherally restricted therapies. Tenvie’s most advanced clinical-stage programs, TNV262, TNV108, and TNV313, are being evaluated in Phase 1 studies, with preliminary data from TNV262 and TNV108 expected by year-end and preliminary data from TNV313 expected in 1H 2027. TNV262 is a fully CNS-penetrant NLRP3 inhibitor being developed for cardiometabolic and neuroinflammatory indications, including obesity, cardiovascular disease, and multiple sclerosis. TNV108 is a peripherally restricted allosteric SARM1 inhibitor being developed for peripheral neuropathies. TNV313 is a fully CNS-penetrant NLRP3 inhibitor being developed for neurologic, cardiometabolic, and ophthalmic diseases. Tenvie is backed by leading life sciences investors, including ARCH Venture Partners, F-Prime Capital, and Mubadala Capital.

Walden Biosciences is a private, clinical-stage biotechnology company focused on developing disease-modifying medicines to treat rare chronic kidney diseases. Walden is applying unique, multi-disciplinary approaches designed to prevent kidney damage and stop disease progression. Walden’s product candidates are well-differentiated from current therapies as they have novel mechanisms of action and are not hemodynamic modulators and are non-immunosuppressive.

Walden’s programs directly target two cell types critical for kidney function: podocytes and proximal tubular cells. Dysfunction of these cells is a critical hallmark of the majority of proteinuric kidney diseases. Walden’s Phase 2 program, WAL0921, is a humanized monoclonal antibody that inhibits urokinase plasminogen activator in both its soluble (suPAR) and membrane bound (uPAR) forms and is being evaluated in a global basket study. Walden’s second most advanced program is a Phase 1 ready small molecule, WAL0623, that is 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. Both programs offer the promise to deliver breakthrough therapies that transform the treatment of kidney disease.

Clinical-stage biotechnology company developing sophisticated, masked, immuno-oncology (I-O) therapies across diverse mechanisms and architectures including bispecifics and T cell engagers (TCE). Clinically validated platform enables engineering of differentiated therapies to optimize the therapeutic index of targets previously limited by severe systemic toxicity. Lead wholly owned program, XTX501, a potential best-in-class bispecific PD-1/masked IL-2, recently received IND clearance, with Phase 1/2 dosing in non-small cell lung cancer expected in 2H 2026 and initial Phase 1 data expected in the 2H 2027. Xilio is also advancing next-generation TCE programs incorporating masking, conditional half-life optimization and co-stimulation to improve tolerability and drive sustained anti-tumor activity. IND submissions anticipated in 2H 2027 for both CLDN18.2 and PSMA+STEAP1 TCE programs. Cash runway extends into 1Q 2028, with potential for extension from strategic partnerships with Gilead and AbbVie and the exercise of outstanding equity warrants.

Genetic medicines company developing targeted delivery technologies, including proprietary targeted lipid nanoparticle (tLNP) and antibody-oligonucleotide conjugate (AOC) platforms, designed to unlock the next generation of sequence-based therapeutics. Advancing a growing pipeline of genetic medicines designed to enable targeted delivery to immune cells, cardiac muscle, skeletal muscle, and other tissues beyond the reach of conventional approaches. The company’s lead program, AERA-109, is entering Phase 1 and is an in vivo CD19 CAR-T therapy for B-cell-mediated autoimmune diseases that leverages targeted lipid nanoparticle technology to generate CAR-T cells directly in patients, with potential as a “pipeline in a product” across multiple autoimmune indications. Aera is also advancing a portfolio of muscle-targeted AOC programs focused on cardiac and skeletal muscle delivery, with the potential to address a broad range of genetic and neuromuscular disorders and overcome current TfR1 limitations. Founded by leaders in RNA therapeutics and genetic medicine.

Biotechnology company developing RNAi therapeutics for severe neurological diseases using proprietary divalent and bispecific siRNA platforms designed to achieve broad CNS distribution, robust target knockdown and durable activity in deep brain regions. Pipeline focused on genetically validated neurological disease targets, initially centered on Huntington's disease (HD) franchise. Lead program ATL-101, HTT-targeting therapy with a potentially best-in-class profile, is expected to enter the clinic by YE26 with initial Ph. 1 safety and biomarker data in HD patients anticipated in 1H27. The company is also advancing potentially first-in-class MSH3-targeting and dual-targeting HTT/MSH3 approaches intended to address complementary drivers of HD onset and progression. Backed by leading life sciences investors and a strategic collaboration with Genentech.

Atavistik Bio is a Cambridge, MA-based clinical stage biotechnology company advancing a pipeline of hematology therapeutics developed through a novel allosteric discovery approach. Atavistik Bio is advancing multiple programs with best-in-class potential, including the lead asset, ATV-1601, an investigational oral allosteric AKT1-selective inhibitor for the treatment of hereditary hemorrhagic telangiectasia (HHT) and advancing a JAK2V617F mutant-selective inhibitor program for myeloproliferative neoplasms. Atavistik Bio is led by an experienced team of drug hunters with a proven track record of developing marketed small molecule therapies and supported by top-tier investors.

Mentari Therapeutics is a Waltham, MA-based biotechnology company developing therapies for the prevention of migraine to deliver freedom from this debilitating and undertreated neurological condition that affects more than 1 billion people globally. Mentari's lead programs target PACAP, a newly validated target that is mechanistically independent from CGRP, one of the first migraine targets to yield clinical and commercial success. Mentari's pipeline includes MT-001, an anti-PACAP monoclonal antibody designed for convenient subcutaneous dosing, and MT-002, an anti-CGRP and anti-PACAP bispecific antibody designed to inhibit these complementary pathways with potential to deliver superior outcomes for people with incomplete response to CGRP-targeted therapies. The company's programs were discovered by Paragon Therapeutics. 

Commercial-stage biopharmaceutical company pioneering myostatin-targeted therapies to transform the treatment of rare neuromuscular diseases. Lead product ISEMBYLD™ (apitegromab-mstn) received FDA approval in September 2026 as the first and only muscle-targeted treatment for children and adults with spinal muscular atrophy (SMA) receiving an SMN2-targeted therapy, with commercial launch now underway in the U.S. The Company is advancing its broader anti-myostatin platform with ongoing Phase 2 OPAL study evaluating apitegromab in infants and toddlers, Phase 2 FORGE study in patients with facioscapulohumeral muscular dystrophy (FSHD), and Phase 1 study of SRK-439, a next-generation subcutaneous myostatin inhibitor.

Clinical-stage genetic medicine company developing precision-targeted, locally delivered gene therapies for monogenic disorders, initially focused on inherited hearing loss. Lead program, SKY-GJB2, is a potential first-in-class gene therapy for GJB2-mediated pediatric hearing loss, the most common genetic cause of deafness. SKY-GJB2 is being evaluated in the Phase 1/2 SONIX trial, with initial data expected by year-end 2026. Skylark’s differentiated platform combines an engineered capsid designed for broad transduction with targeted microdose delivery, cell-specific expression control, and the potential for durable therapeutic benefit. Additional pipeline programs include SKY-AS for Angelman syndrome and SKY-PEN for SLC26A4-related hearing loss, both in IND-enabling studies.

StradBio is developing next-generation ADCs and immunotherapies for cancer and inflammatory diseases. Its lead asset, SB-230, is an anti-CD30 ADC with a Topo-1 payload and has demonstrated encouraging clinical activity, including 28 partial and 13 complete responses in 53 heavily pretreated lymphoma patients in the dose-escalation portion of a Phase 1 study. The pipeline also includes SB-225, an affinity-modified anti-CD25 ADC with an MMAE payload being developed for lymphoma, leukemias, and solid tumors. In addition, StradBio is advancing Daiad-2/15, a PD-1-targeted highly potent IL-2/IL-15 agonist designed to enhance antitumor immunity while improving tolerability, along with a broader portfolio of Daiad-based programs for autoimmune and metabolic diseases. The company plans to provide updated clinical data and development plans for SB-230 and other pipeline assets. StradBio completed a Series A financing in October 2026 and is led by CEO Jonathan Drachman, former Chief Medical Officer of Seagen and former CEO of Neoleukin Therapeutics.

Clinical-stage oncology company focused on expanding the breakthrough potential of targeted radiopharmaceuticals to large patient populations. The company is advancing a multi-asset pipeline driven by its novel miniprotein radioconjugate platform and patient-centric end-to-end manufacturing and supply chain capabilities. Miniproteins selectively deliver radioisotopes to tumors, enabling high tumor penetration, uptake, and retention while rapidly clearing from normal tissues, thereby enhancing the therapeutic potential of radiopharmaceuticals. The first two programs represent potential first-in-class, multi-tumor, and multi-indication opportunities. Lead program AKY-1189 is a Nectin-4-targeted miniprotein radioconjugate in a Phase 1b clinical trial enrolling patients with locally advanced or metastatic urothelial carcinoma (mUC) and other Nectin-4-expressing solid tumors, with preliminary data expected in 1Q'27. AKY-2519 is a B7-H3-targeted miniprotein radioconjugate being evaluated in two Phase 1b trials: the first enrolling patients with mCRPC, with preliminary data expected in 2027, and the second enrolling patients with NSCLC and other B7-H3-expressing tumors.

AdvanCell (Private) is a clinical-stage radiopharmaceutical company developing targeted alpha therapies for the treatment of cancer. Leveraging a vertically integrated model spanning discovery, development, and manufacturing, the company is advancing a pipeline of novel radiopharmaceutical candidates, including lead program ADVC001 for the treatment of prostate cancer. In 2026, AdvanCell completed an oversubscribed $315 million Series D financing to support continued clinical development and the expansion of its global manufacturing infrastructure. Backed by leading healthcare investors and strategic partners, AdvanCell is focused on establishing a new standard of care through targeted alpha therapies for patients with difficult-to-treat cancers.

Clinical-stage biotech company engineering the next generation of RNAi therapeutics. Proprietary City Platform combines novel RNAi trigger engineering, including cityRNA triggers designed for extrahepatic tissues, tissue-selective delivery technologies, and genetics-driven target selection with the aim of systematically developing potentially best-in-class RNAi therapeutics and delivering potent, specific, and durable silencing of disease-causing proteins in and beyond the liver. Pipeline includes CITY-FXI for thromboembolic diseases in Phase 1 with updated data expected in late 2026, Phase 1-ready CITY-RBP4 for Stargardt disease and CITY-TFR2 for anemia of chronic disease, both with initial data expected in mid-2027, and goal of filing 1–2 new INDs or IND equivalents annually. Founded by pioneering executives and scientists in RNAi therapeutics, City is advancing differentiated wholly owned programs, with goal of expanding into additional tissues, and leveraging partnerships with Bausch + Lomb and Biogen in ophthalmology and CNS diseases.

Clinical-stage genetic medicines company pioneering a novel class of RNA-editing therapeutics designed to repair disease-causing genetic variants or introduce protective variants. Leading RNA-editing RESTORE+ platform is purpose-built to design potent, convenient, and scalable therapeutics without the risks of off-target editing and permanent changes to DNA to deliver precision therapies for genetically defined diseases. Lead candidate AIR-001 is a GalNAc-conjugated oligonucleotide targeting the treatment of alpha-1 antitrypsin deficiency (AATD), where RNA editing has been clinically de-risked. AIR-001 is designed to correct the pathogenic PiZ mutation at the RNA level, restoring the production of functional M-AAT protein. Preclinical studies demonstrated potential best-in-class efficacy, achieving up to 59% precise RNA editing in vivo and restoring 39 µM of functional M-AAT in serum. Enrollment ongoing in the Phase 1 RepAIR1 trial evaluating safety and efficacy of AIR-001 in AATD patients, with interim data expected in 1H 2027. Backed by top-tier investor syndicate, including ARCH Venture Partners, Forbion, Nextech, RTW Investments, and Venrock.

Clinical-stage biotechnology company discovering and developing a broad pipeline of allogeneic gamma delta CAR T-cell therapies, and differentiated in-vivo CAR-T therapies with the potential to redefine treatment across autoimmune diseases, hematologic malignancies, and solid tumors. Positive Phase 1 data for lead program prulacabtagene leucel (prula-cel; formerly ADI-001), reported in September 2026, demonstrated treatment-free remissions and a favorable safety profile at 12 months, including 54% DORIS remission in SLE patients with or without lupus nephritis (LN) and 50% CRR in LN, supporting plans to initiate pivotal start-up activities in 4Q26. Broader pipeline includes ADI-212, a gene-edited, armored cell therapy candidate targeting PSMA for the treatment of prostate cancer, with Phase 1 study planned for 4Q26 initiation, as well as a next-generation in vivo CAR-T platform targeting hematologic and solid tumors, with first candidate expected to enter the clinic in 2H 2027.

Achieve Life Sciences is a late-stage specialty pharmaceutical company focused on the global development and commercialization of cytisinicline as a treatment for nicotine dependence. Achieve’s New Drug Application (NDA) for cytisinicline for smoking cessation in adults is supported by two successfully completed Phase 3 studies and an open-label long-term safety study. The company anticipates re-submitting the NDA for cytisinicline for smoking cessation in the fourth quarter of 2026, with approval anticipated in the first half of 2027. Achieve has also completed a Phase 2 study of cytisinicline in nicotine e-cigarette cessation, conducted an end-of-Phase 2 meeting with the FDA, and has received Breakthrough Therapy designation for the vaping cessation indication.

Direct Biologics is a biotechnology company advancing research in investigational secretome-based products, including extracellular vesicles (EVs), derived from adult bone marrow mesenchymal stem cells (MSCs).

Guided by its core values of Safety, Innovation, and Quality, Direct Biologics is developing investigational secretome-based products being studied in clinical research under current Good Manufacturing Practice (cGMP) manufacturing standards. The Company combines scientific research, quality-focused manufacturing, and clinical investigation to support the advancement of its development programs.

Direct Biologics’ lead program is currently in a Phase 3 clinical trial for acute respiratory distress syndrome (ARDS), a life-threatening condition characterized by severe lung inflammation and respiratory failure that affects hundreds of thousands of patients globally and represents a significant unmet medical need with limited therapeutic options. In addition to its ARDS program, the Company's clinical development pipeline includes investigational programs studying perianal fistulizing Crohn's disease and medically refractory Crohn's disease.

Direct Biologics is committed to advancing scientific understanding of immune and inflammatory responses and contributing to ongoing research in regenerative medicine.

Direct Biologics develops investigational products that have not been approved by the U.S. Food and Drug Administration. Their safety and effectiveness have not been established.