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BioGene Therapeutics Inc. Appoints Dr. Francis Tavares, PhD., as Chief Technology Officer
Newsfile· 2025-08-20 11:00
Company Overview - PreveCeutical Medical Inc. is a health sciences company focused on developing innovative preventive and curative therapies using organic and nature-identical products [5] - The company has five research and development programs targeting diabetes, obesity, and other health issues, including dual gene therapy and non-addictive analgesic peptides [5] Leadership Appointment - BioGene Therapeutics Inc., a wholly owned subsidiary of PreveCeutical, appointed Dr. Francis Tavares as Chief Technology Officer effective August 19, 2025 [1][4] - Dr. Tavares has extensive experience in the biopharmaceutical industry, previously serving as President, CEO, and Founder of ChemoGenics BioPharma, and has a strong background in drug discovery and medicinal chemistry [2][3] Dr. Tavares' Contributions - Dr. Tavares has a PhD in Organic Chemistry and has led the development of significant drug candidates, including Trilaciclib and Lerociclib, demonstrating a successful track record in advancing drug candidates to clinical trials [3] - His appointment is expected to enhance BioGene's capabilities in drug discovery and product commercialization, benefiting both patients and investors [4] BioGene Therapeutics Inc. Overview - BioGene is focused on advancing innovative therapies in metabolic health and gene-based treatments, with a subsidiary in Australia that leverages a 43.5% R&D tax cashback incentive [6] - The company is engaged in research aimed at addressing the global diabetes and obesity crisis through gene therapy-based approaches [6]
BioGene Therapeutics Inc. Appoints Dr. Kamal Albarazanji as Senior Director of Metabolic Research
Newsfile· 2025-08-19 11:00
Core Insights - BioGene Therapeutics Inc. has appointed Dr. Kamal Albarazanji as Senior Director of Metabolic Research effective August 18, 2025, to enhance its research capabilities in metabolic health [1][4]. Company Overview - PreveCeutical Medical Inc. is a health sciences company focused on developing innovative preventive and curative therapies using organic and nature-identical products, with five ongoing research and development programs targeting diabetes, obesity, and other health issues [5]. - BioGene Therapeutics Inc. is a Texas-based life sciences company dedicated to advancing therapies in metabolic health and gene-based treatments, with a subsidiary in Australia benefiting from a 43.5% R&D tax cashback incentive [7]. Dr. Kamal Albarazanji's Background - Dr. Albarazanji has extensive expertise in in vivo pharmacology, target validation, and translational research, having previously worked on programs related to metabolic diseases and cardiovascular health at a leading pharmaceutical company [2]. - He has a notable history at SmithKline Beecham (now GSK), contributing to significant advancements in hypertension and obesity treatments [2]. - Dr. Albarazanji holds numerous patents and has authored over 20 peer-reviewed manuscripts in prestigious journals, showcasing his prolific research contributions [3]. Strategic Importance - The appointment of Dr. Albarazanji is expected to accelerate BioGene's pipeline of innovative therapeutics, aligning with the company's mission to address unmet needs in obesity and diabetes [4]. - Dr. Albarazanji's vision and expertise are anticipated to enhance the company's research programs and lead to meaningful outcomes for patients [4].
REGENXBIO Announces FDA Review Extension of BLA for RGX-121 to Treat Patients with MPS II
Prnewswire· 2025-08-18 21:00
Core Viewpoint - REGENXBIO Inc. announced an extension of the FDA review timeline for its Biologics License Application (BLA) for RGX-121, a treatment for Mucopolysaccharidosis II (MPS II), from November 9, 2025, to February 8, 2026 [1] Group 1: FDA Review and Clinical Data - The extension follows the submission of long-term clinical data for all patients in the pivotal study of RGX-121, which included 13 patients, in response to an FDA information request [2] - Positive 12-month clinical data are consistent with previously submitted biomarker and neurodevelopmental data and will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM) in September 2025 [2] - The FDA completed a pre-license inspection and bioresearch monitoring inspection for the RGX-121 BLA with no observations and no safety-related concerns raised during the review [3] Group 2: Company Statements and Designations - The President and CEO of REGENXBIO emphasized the urgent need for a therapeutic option for boys with Hunter syndrome and expressed confidence that commercial launch plans remain on track [4] - RGX-121 has received multiple designations from the FDA, including Orphan Drug Product, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) [4] Group 3: Product and Disease Overview - RGX-121 is a potential one-time AAV therapeutic designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system, potentially providing a permanent source of the I2S protein [5] - MPS II, or Hunter Syndrome, is a rare disease caused by a deficiency in the lysosomal enzyme I2S, leading to significant medical needs, particularly for neurological manifestations [6] Group 4: Future Plans and Market Potential - If approved, RGX-121 would be the first and only commercially available therapy designed to address the underlying genetic cause of Hunter syndrome [8] - REGENXBIO plans to present updated pivotal data during the ICIEM meeting in September 2025 [8]
X @Bloomberg
Bloomberg· 2025-08-18 16:10
Industry Focus - The report focuses on a controversial gene therapy for boys with a rare muscle disease [1] - The report details how parents influenced the return of the gene therapy to the market [1]
MeiraGTx Reports Second Quarter 2025 Financial and Operational Results
GlobeNewswire News Room· 2025-08-14 12:00
Core Insights - MeiraGTx Holdings plc has made significant progress in its clinical programs, including alignment with the FDA on the Phase 2 study of AAV-hAQP1 for radiation-induced xerostomia, which is expected to support a Biologics License Application (BLA) filing by late 2026 [1][2] - The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD, a gene therapy for Parkinson's disease, indicating its potential to address serious conditions [1][5] - The company is preparing to file for Marketing Authorization Approval (MAA) in the UK and BLA in the US for AAV-AIPL1, targeting LCA4, with submissions expected in Q4 2025 [1][2] Regulatory Developments - The company has had productive interactions with the FDA and MHRA regarding multiple late-stage clinical programs and manufacturing processes [2] - AAV-GAD has shown significant disease-modifying effects in clinical trials, with plans to initiate a Phase 3 study later this year [1][6] - The FDA has completed a Good Clinical Practice inspection of the AAV-GAD bridging study, resulting in a clean inspection with no observations [6][8] Financial Performance - As of June 30, 2025, the company reported cash and cash equivalents of approximately $32.2 million, a decrease from $101.0 million a year earlier [18][19] - Service revenue increased to $3.7 million for Q2 2025, up from $0.3 million in Q2 2024, attributed to progress in process performance qualification services [19][22] - The net loss attributable to ordinary shareholders for Q2 2025 was $38.8 million, compared to a net loss of $48.6 million in Q2 2024, indicating an improvement in financial performance [28][32] Clinical Programs and Collaborations - The company is advancing its late-stage clinical programs, including AAV-hAQP1 for xerostomia and AAV-GAD for Parkinson's disease, with pivotal data readouts expected in late 2026 [1][2] - MeiraGTx is collaborating with Hologen to develop AAV-GAD, with a joint venture established to finance the program and utilize AI for data analysis [6][8] - The company is also preparing to initiate first-in-human studies using its riboswitch platform by the end of 2025 [2][20] Manufacturing and Technology - MeiraGTx has developed comprehensive in-house manufacturing capabilities, with facilities in the UK and Ireland holding necessary regulatory authorizations [15][17] - The company is focusing on optimizing its riboswitch gene regulation technology for various therapeutic applications, including metabolic diseases and chronic pain [29][30] - The manufacturing process for AAV2-hAQP1 will be in-house, ensuring consistency across different indications [5][8]
Opus Genetics Announces Financial Results for Second Quarter 2025 and Provides Corporate Update
Globenewswire· 2025-08-13 20:30
Core Insights - Opus Genetics has achieved significant progress in its clinical pipeline, including positive clinical data for OPGx-LCA5 and FDA's RMAT designation, indicating strong potential for vision restoration therapies [1][2][5] - The company reported positive topline results from Phase 3 trials for Phentolamine Ophthalmic Solution 0.75%, which could provide new treatment options for patients with vision challenges [1][2][13] - Financial results for Q2 2025 show an increase in collaboration revenue and a net loss reduction compared to the same period in 2024, indicating improved financial performance [9][12][22] Pipeline Updates - OPGx-LCA5, a gene therapy for Leber Congenital Amaurosis, has shown sustained visual function improvements in adult patients, with initial pediatric data indicating positive outcomes [1][5] - OPGx-BEST1 is on track to enter Phase 1/2 trials in H2 2025, targeting bestrophin-1 related inherited retinal disease [1][4][14] - Non-dilutive funding has been secured to support the advancement of multiple early-stage gene therapy programs, including OPGx-RDH12 and OPGx-MERTK [1][13] Financial Performance - As of June 30, 2025, Opus Genetics reported cash and cash equivalents of $32.4 million, expected to fund operations into the second half of 2026 [8] - License and collaboration revenue for Q2 2025 totaled $2.9 million, up from $1.1 million in Q2 2024, driven by collaboration with Viatris, Inc. [9] - General and administrative expenses increased to $5.8 million in Q2 2025 from $3.4 million in Q2 2024, primarily due to higher legal and patent-related costs [10] Upcoming Milestones - The company plans to report three-month pediatric data from the OPGx-LCA5 trial in Q3 2025 and initiate enrollment for OPGx-BEST1 in H2 2025 [14] - A supplemental New Drug Application (sNDA) for Phentolamine is expected to be submitted in the second half of 2025 [14] - Enrollment for the LYNX-3 Phase 3 trial targeting nighttime visual disturbances is anticipated to begin in H2 2025 [14]
Tenaya Therapeutics (TNYA) Earnings Call Presentation
2025-08-13 20:00
Pipeline and Programs - Tenaya Therapeutics has 3 clinical-stage programs focused on heart disease[9] - TN-201, a gene therapy for MYBPC3-associated HCM, is in Phase 1b/2 clinical trials with data readouts expected in Q4 2025[10, 12] - TN-401, a gene therapy for PKP2-associated ARVC, is in Phase 1b clinical trials with initial data including safety and biopsy results expected in 2025[10, 94] - TN-301, a small molecule HDAC6 inhibitor for HFpEF, has completed Phase 1 trials and is seeking a partner for further development[10, 99, 104] TN-201 for MYBPC3-associated HCM - MYBPC3-associated HCM affects an estimated 120,000 people in the U S alone[22] - Interim Cohort 1 data for TN-201 showed the therapy was well-tolerated at 3E13 vg/kg dose, with no cardiotoxicities and manageable liver enzyme elevations[17, 40] - All three patients in Cohort 1 improved from NYHA class II/III to NYHA class I, and elevated troponin levels dropped by 60% in two patients into normal ranges[19] - TN-201 DNA in cardiac biopsy surpassed preclinical efficacy threshold and compares favorably to peer, with long-term stability achieved[42] - TN-201 RNA expression increased by as much as 13-fold from week 8 to Week 52[46] TN-401 for PKP2-associated ARVC - PKP2-associated ARVC is estimated to affect over 70,000 people in the U S [75] - Preclinical studies showed that a single 3E13 vg/kg dose of TN-401 in a KO mouse model reversed hallmarks of the disease and extended survival[91] - RIDGE natural history study shows that over 80% of ARVC patients continue to experience high PVC count despite standard of care treatments[87]
4D Molecular Therapeutics (FDMT) FY Conference Transcript
2025-08-13 19:30
Summary of 4D Molecular Therapeutics (FDMT) FY Conference Call Company Overview - **Company**: 4D Molecular Therapeutics (FDMT) - **Industry**: Gene Therapy, specifically focusing on ophthalmology and retinal diseases - **Key Product**: 4D-150, a gene therapy for wet age-related macular degeneration (AMD) currently in Phase 3 trials [3][4] Core Points and Arguments Gene Therapy Platform - 4D has developed a next-generation gene therapy platform utilizing directed evolution technology to create targeted vectors for specific tissues [3] - The company has been operational for over ten years, focusing on genetic medicines [3] Product Development - **4D-150**: The most advanced program, currently in Phase 3 for wet AMD, with promising Phase 1 and 2 data showing efficacy and safety [4][10] - The therapy uses a proprietary intravitreal vector (R100) designed for single injection durability, expressing aflibercept (Eylea) [6][7] Efficacy and Safety - Significant reduction in treatment burden and high rates of injection-free patients observed across various patient populations [7][32] - Safety profile of 4D-150 is strong, with no major signs of intraocular inflammation or severe adverse events reported in recent studies [10][33] Market Need and Competitive Landscape - Current anti-VEGF therapies require frequent injections, leading to adherence issues and potential vision loss over time [19][20] - 4D-150 aims to provide continuous delivery of aflibercept, addressing the treatment burden and improving long-term outcomes [20][21] Commercialization Strategy - The company anticipates that 4D-150 will become a backbone therapy in the treatment of wet AMD, with potential for use in treatment-naive patients as well [29][36] - Initial commercialization may focus on patients with high unmet needs, gradually expanding to a broader patient population as physicians gain confidence in the therapy [36][38] Important Data Points - In the Phase 2b PRISM trial, 60% of patients were injection-free at one year, with 80% injection-free rates in more recently diagnosed patients [34][42] - Upcoming catalysts include longer-term durability data expected in Q4 2025 and updates on enrollment progress for ongoing trials [46][47] Underappreciated Aspects - The significant unmet need for durable therapies in the retinal space is validated by recent surveys indicating high excitement among retina specialists for gene therapy [50][51] - The potential for 4D-150 to provide long-term treatment burden reduction is a compelling value proposition for both patients and payers [25][51] Conclusion - 4D Molecular Therapeutics is positioned to address significant gaps in the treatment of wet AMD through its innovative gene therapy approach, with strong efficacy and safety data supporting its commercial potential [52]
Candel Therapeutics (CADL) FY Conference Transcript
2025-08-13 19:00
Summary of Candel Therapeutics (CADL) FY Conference Call - August 13, 2025 Company Overview - **Company**: Candel Therapeutics (CADL) - **Industry**: Biotechnology, specifically focused on cancer immunotherapy Key Points and Arguments Vaccine Approach - Candel's approach involves a novel vaccine strategy that immunizes patients against their own tumors without needing to identify specific antigens [3][4] - Utilizes a replication-defective adenovirus to deliver the HSV thymidine kinase gene, leading to localized enzyme expression and subsequent tumor cell death [4][5] - The process induces a strong pro-inflammatory response, creating optimal conditions for T cell activation against tumors [5][6] Pipeline Focus - Current focus on three main indications: - Early localized nonmetastatic prostate cancer - Borderline resectable pancreatic cancer - Therapy-resistant non-small cell lung cancer (NSCLC) [7][8] Prostate Cancer Data - Positive Phase 3 trial results with a primary endpoint of disease-free survival, showing a 30% improvement in disease-free survival rates [10][12] - Secondary endpoint showed a 38% improvement in prostate cancer-specific disease-free survival [13] - Plans to submit a Biologics License Application (BLA) by 2026 [9][10] Commercial Launch Preparation - Scaling up commercial manufacturing with partner SAFC in California [16][17] - Positive feedback from urologists and radiation oncologists regarding the adoption of CAN 2409 in combination with standard care [18][20] - Initial payer feedback has been favorable, indicating potential cost savings for healthcare systems [21] Non-Small Cell Lung Cancer (NSCLC) Data - Focus on patients with unresectable stage 3 or stage 4 NSCLC who have failed standard treatments [23] - Median overall survival of over 24 months in treated patients, doubling the expected survival [24] - Fast track designation received from the FDA based on these results [26][27] Pancreatic Cancer Data - Conducted a small randomized trial showing a median overall survival of over 32 months compared to 12.5 months in the control group [34] - Fast track and orphan drug designations received from the FDA and EMA [35] Manufacturing and Capacity - Manufacturing process involves replication-defective adenoviruses, similar to COVID-19 vaccines, with established industry capacity [38][39] - Product stability confirmed for over ten years under refrigeration [40] Future Directions - Candel is preparing for a potential registrational Phase 3 trial in therapy-resistant NSCLC and pancreatic cancer [27][35] - Ongoing exploration of CAN 3110, a next-generation oncolytic virus for glioblastoma, showing promising early results [42][45] Financial Position - Current cash runway extends into Q1 2027, with upcoming data announcements expected [53] Additional Important Information - Candel emphasizes the importance of a strong scientific rationale and unmet clinical needs in prioritizing its pipeline [36] - The company aims to advance multiple programs in parallel to maximize commercial value [37]
Tenaya Therapeutics (TNYA) FY Earnings Call Presentation
2025-08-12 15:30
Clinical Programs & Data - Tenaya Therapeutics has 3 clinical-stage programs focused on heart disease [9] - TN-201 for MYBPC3-associated HCM plans data release including longer-term follow-up for Cohort 1 (3E13 vg/kg) and initial safety and biopsy results for Cohort 2 (6E13 vg/kg) in Q4'25 [15] - TN-401 for PKP2-associated ARVC plans data release will include initial Cohort 1 (3E13 vg/kg) data focused on safety and biopsy results in Q4'25 [16] - Interim Cohort 1 TN-201 data showed improvements in ≥ 1 measures of hypertrophy in two of three patients [17] Disease & Patient Populations - MYBPC3-associated HCM is estimated to affect 120,000 people in the U S alone [22] - Approximately 57% of identified genetic variants underlying familial HCM are MYBPC3 mutations [22] - Over 30% of genetic variants underlying childhood-onset HCM are MYBPC3 mutations [22] - PKP2-associated ARVC is estimated to affect >70,000 people in the U S [75] - In ARVC patients, >15% of heart-related deaths in patients < 35 are due to ARVC [75] - In ARVC patients, 23% of ARVC patients present with sudden cardiac death [75] - In ARVC patients, 40% of ARVC patients carry pathogenic PKP2 mutations [75] Capabilities & Milestones - Tenaya has cGMP AAV manufacturing scale achieved; 1000L clinical supply for TN-201 and TN-401 ready [115]