✨ Onyx Summary Affinia Therapeutics CEO Rick Modi will participate in a panel on in vivo gene therapies for genetic cardiac diseases at Chardan’s 9th Annual Genetic Medicines Conference on Oct. 21 and hold one-on-one investor meetings at the Jefferies Global Healthcare Conference in London on Nov. 17–20. The
✨ Onyx Summary Rocket Pharmaceuticals announced that the U.S. FDA has accepted its resubmitted BLA for KRESLADI™ (marnetegragene autotemcel; marne-cel), an investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I), setting a PDUFA date of March 28, 2026. The filing is supported by Phase 1/2 data showing 100% survival
✨ Onyx Summary Verily has entered a multi-year strategic collaboration with UCHealth, the University of Colorado Anschutz, and RefinedScience to advance precision medicine through AI-driven data integration and analytics on Verily’s healthcare technology platform. The partnership will create AI-ready biomedical data pipelines, develop novel models across major therapeutic areas, and
✨ Onyx Summary Excellergy, a Palo Alto–based biotechnology company, launched with a $70 million Series A financing led by Samsara BioCapital to advance its first-in-class Effector Cell Response Inhibitors (ECRIs), a novel therapeutic platform targeting IgE-mediated allergic disease. The funding will support the company’s move into clinical trials in
✨ Onyx Summary Geron Corporation announced multiple leadership changes to align with its commercial growth strategy, including the appointment of Ahmed ElNawawi as EVP and Chief Commercial Officer, alongside new senior hires in technical operations, investor relations, and portfolio management. These appointments strengthen Geron’s leadership capabilities as it drives commercialization
✨ Onyx Summary Avidity Biosciences completed a positive pre-BLA meeting with the U.S. FDA for delpacibart zotadirsen (del-zota), its investigational Duchenne muscular dystrophy (DMD44) therapy, and now plans to file the BLA in Q1 2026 to include additional CMC data. The company’s first planned BLA submission marks a key
✨ Onyx Summary Seer, Inc. announced its participation at the 2025 American Society of Human Genetics Annual Meeting, where it will showcase how its Proteograph® Product Suite enables integration of proteomics and genomics to advance precision medicine. The company will host a featured CoLab session and support multiple scientific presentations demonstrating
✨ Onyx Summary Rocket Pharmaceuticals granted inducement equity awards to newly appointed Chief Medical Officer Dr. Syed Rizvi, consisting of stock options and restricted stock units totaling approximately 729,765 shares. The awards, approved under Nasdaq’s inducement grant rule, align long-term incentives with Rocket’s strategic growth as it advances
✨ Onyx Summary Lexicon Pharmaceuticals hosted a policy roundtable in Washington, DC, bringing together clinicians, patient advocates, and industry experts to discuss expanding the scope of the federal “Alternatives to PAIN Act” to include chronic pain sufferers, beyond its current focus on acute and post-surgical pain. CEO Mike Exton emphasized the
✨ Onyx Summary Lexicon Pharmaceuticals convened a policy roundtable in Washington, D.C., with clinicians, advocates, and patients to discuss expanding the Alternatives to PAIN Act to include chronic pain patients under Medicare Part D coverage for non-opioid treatments. The company plans to publish a White Paper summarizing insights from the
✨ Onyx Summary Affinia Therapeutics closed a $40 million Series C financing led by NEA with participation from Eli Lilly and existing investors to advance its lead gene therapy candidate AFTX-201 for BAG3 dilated cardiomyopathy (DCM). The proceeds will support IND submission in Q4 2025 and initiation of the Phase 1/
✨ Onyx Summary Aspect Biosystems Chief Scientific Officer Sam Wadsworth will present at the 2025 Cell & Gene Meeting on the Mesa on October 7 in Phoenix, highlighting the company’s advancements in regenerative medicine. The annual conference, organized by the Alliance for Regenerative Medicine, gathers global leaders in cell and
✨ Onyx Summary Avidity Biosciences will present new one-year data on delpacibart zotadirsen (del-zota) for Duchenne muscular dystrophy (DMD44) at the 30th Annual World Muscle Society Congress, showing reversal of disease progression and improvements across functional measures from its EXPLORE44® and EXPLORE44-OLE™ trials. The presentations, featuring leading clinicians, reinforce del-zota’s
✨ Onyx Summary Phathom Pharmaceuticals appointed Sanjeev Narula as Chief Financial and Business Officer, bringing over two decades of financial leadership experience from Intra-Cellular Therapies, Viatris, and Pfizer. His appointment strengthens Phathom’s executive team as the company advances commercialization of its GI franchise, VOQUEZNA, and pursues its strategy toward profitability
✨ Onyx Summary MassBio will host the 2025 Patient Advocacy Summit on November 6 at the MassBioHub, themed “Purpose, In Spite of Pressure,” highlighting how patients and advocates drive progress across the healthcare and drug development landscape. The event will feature a keynote by Yelak Biru and Fatima Scipione on the
✨ Onyx Summary Perspective Therapeutics announced that the first patient has been treated in the second cohort of its Phase 1/2a trial evaluating [212Pb]PSV359, a targeted alpha radiopharmaceutical for solid tumors expressing FAP-α. The study advances dose escalation to 5.0 mCi following favorable safety review at lower doses,
