MicroStockHub/iStock via Getty Images Listen here or on the go via Apple Podcasts and Spotify How to trade and invest in the biotech space with ROTY Biotech Community's Jonathan Faison (0:35). Making money in biotech (5:40). Managing sector risk (9:20). Geron - a stock he never thought he'd own (17:10). Transcript Rena Sherbill: Jonathan Faison from ROTY Biotech Community on Seeking Alpha. That's the investing group you run. Jonathan, welcome back to Investing Experts. Jonathan Faison: Thank you, Ren ...

Key Takeaways Bayer and Kumquat begin a phase I study of KQB548 for KRAS G12D-mutated tumors.The study will assess the safety and early efficacy in pancreatic, colorectal and lung cancers.KQB548 is the lead program from Bayer and Kumquat's exclusive oncology collaborationBayer (BAYRY) , along with San Diego, CA-based, privately held company, Kumquat Biosciences, announced the initiation of a phase I study evaluating their investigational inhibitor, KQB548 (BAY 3771249), which is being developed for treating ...

, /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present interim data from the Phase II ALTITUDE trial evaluating suprachoroidal delivery of surabgene lomparvovec (ABBV-RGX-314, sura-vec) for the treatment of diabetic retinopathy (DR) at the American Academy of Ophthalmology 2025 Annual Meeting. Sura-vec, developed in collaboration with AbbVie, is an investigational one-time gene therapy and potential first-in-class treatment for wet age-related macular degeneration (wet AMD) an ...

Key Takeaways BlueRock posted 36-month data showing bemdaneprocel remains well tolerated in Parkinson's patientsF-Dopa imaging suggests that transplanted cells survived and engrafted after immunosuppression ended. High-dose patients saw a 17.9-point MDS-UPDRS motor score drop, showing clinically meaningful progress.Bayer AG’s (BAYRY) wholly owned, independently operated subsidiary BlueRock Therapeutics LP announced positive 36-month data from an early-stage study of bemdaneprocel, an investigational cell th ...

HYOGO, Japan--(BUSINESS WIRE)--JCR presented non-clinical data that demonstrate the ability of its proprietary JUST-AAV platform to achieve efficient delivery of AAV gene therapy. ...

Lexeo Therapeutics Conference Call Summary Company Overview - **Company**: Lexeo Therapeutics (NasdaqGM:LXEO) - **Focus**: Development of LX2006 for the treatment of Friedreich ataxia cardiomyopathy Key Industry Insights - **Regulatory Update**: Discussions with the FDA regarding a potential accelerated approval pathway for LX2006 are ongoing, with the FDA open to a BLA submission that pools clinical data from ongoing Phase 1-2 studies and an upcoming pivotal study [2][4] - **Clinical Data**: New interim clinical data from two ongoing Phase 1-2 studies show promising results in safety and efficacy for LX2006 [2][4] Core Points and Arguments - **FDA Feedback**: The FDA has agreed to evaluate the co-primary endpoint of left ventricular mass index (LVMI) at an earlier time point than 12 months, potentially shortening the pivotal study duration [4][11] - **Clinical Improvements**: - A 23% mean improvement in LVMI observed at 12 months, with an 18% mean improvement at six months, exceeding the FDA's threshold of 10% for the pivotal study [5][21] - Clinically meaningful improvements in the modified Friedreich ataxia rating scale (MFARS) were also reported [5][27] - **Safety Profile**: LX2006 has been generally well tolerated across all dose cohorts, with no significant adverse events reported [5][18] Additional Important Information - **Patient Population**: Friedreich ataxia affects approximately 5,000 people in the U.S. and 15,000 globally, with cardiac complications being the leading cause of death [6][7] - **Mechanism of Action**: LX2006 aims to treat the root cause of the disease by restoring frataxin, a protein that is deficient in individuals with Friedreich ataxia [8][9] - **Manufacturing Changes**: Lexeo has transitioned to an optimized SF9 baculovirus manufacturing platform, which is expected to produce high-yield, high-quality vector with a low empty capsid ratio [10][11] - **Future Plans**: The pivotal study is expected to be initiated in the first half of 2026, pending finalization of the protocol with the FDA [13][44] Summary of Clinical Data - **Participant Data**: - 17 participants treated to date, with 6 having abnormal LVMI at baseline [14][15] - Improvements in cardiac biomarkers observed, with reductions in LVMI and troponin levels [20][21][23] - **Functional Improvement**: Evidence of functional improvement in MFARS scores, indicating better daily living activities for participants [27][29] Conclusion - Lexeo Therapeutics is making significant progress in the development of LX2006 for Friedreich ataxia cardiomyopathy, with promising clinical data and a favorable safety profile. The company is actively engaging with the FDA to expedite the approval process, aiming to address the urgent unmet need for effective treatments in this patient population [35][77]

LONDON and NEW YORK, Oct. 07, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the Company will exhibit two posters at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress, which is being held from October 7-10, 2025, in Seville, Spain. The posters are available on the Posters and Publications page of the Company’s website. The details of the poster presentations are as follows: Poster Numb ...

RJVA-002 achieved ~30% weight loss at five weeks in a translational obesity model; weight loss not yet plateauedPreclinical results show that a single point-in-time treatment with RJVA-002 has the potential to achieve significant weight loss that can match or exceed best-in-class chronic drug therapy BURLINGTON, Mass., Oct. 07, 2025 (GLOBE NEWSWIRE) -- Fractyl Health, Inc. (Nasdaq: GUTS) (the Company), a metabolic therapeutics company focused on pattern-breaking approaches to treat the root causes of obesit ...

U.S. Food and Drug Administration (FDA) open to pooling data from ongoing Phase I/II studies of LX2006 with pivotal data to support a Biologics License Application (BLA) for Accelerated Approval Interim clinical data show sustained or deepening improvements in the majority of participants across both cardiac and neurologic measures of Friedreich ataxia Participants with abnormal left ventricular mass index (LVMI) at baseline achieved 18% mean reduction in LVMI at 6 months and 23% mean reduction at 12 months ...

, /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the completion of enrollment in the ATMOSPHERE and ASCENT pivotal studies evaluating surabgene lomparvovec (sura-vec, ABBV-RGX-314) in wet age-related macular degeneration (wet AMD) using subretinal delivery. "Completing enrollment in this large, global pivotal program is an exciting milestone in our efforts to deliver sura-vec to patients as the potential first gene therapy for wet AMD," said Steve Pakola, M.D., Chief Medical Officer, REGENXBI ...