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MeiraGTx Announces Two Posters at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress
Globenewswire· 2025-10-07 12:00
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 ...
MeiraGTx Stock: Hologen Partnership Makes Parkinson’s Gene Therapy Vital (NASDAQ:MGTX)
Seeking Alpha· 2025-09-25 07:48
MeiraGTx Holdings plc (NASDAQ: MGTX ) is a genetic medicines biotech with a late-stage pipeline and capabilities for end-to-end GMP manufacturing. Its main drug candidates are AAV-AIPL1 for congenital blindness, which is working toward exceptional-circumstances filings and has a plannedMy name is Myriam Hernandez Alvarez. I received the Electronics and Telecommunication Engineering degree from the Escuela Politecnica Nacional, Quito, Ecuador, the M.Sc. degree in computer science from Ohio University, Athens ...
Krystal Biotech to Present at Cantor Global Healthcare Conference 2025
Globenewswire· 2025-09-02 12:00
Company Participation - Krystal Biotech, Inc. will participate in the Cantor Global Healthcare Conference 2025 on September 4, 2025, in New York [1] - Krish S. Krishnan, Chairman and CEO, will engage in a fireside chat at 10:20 am ET and host investor meetings throughout the day [1] Webcast Information - A webcast of the presentation will be available starting at 10:20 am ET on September 4, 2025, and will be posted on the Company's website [2] Company Overview - Krystal Biotech, Inc. is a fully integrated, commercial-stage global biotechnology company focused on genetic medicines for diseases with high unmet medical needs [3] - The Company's first commercial product, VYJUVEK®, is the first-ever redosable gene therapy and the first genetic medicine approved in the US, Europe, and Japan for dystrophic epidermolysis bullosa [3] - The Company is advancing a robust pipeline of investigational genetic medicines in various fields including respiratory, oncology, dermatology, ophthalmology, and aesthetics [3]
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]
Beam Therapeutics Reports Second Quarter 2025 Financial Results and Provides Update on BEAM-302 Development Progress in Alpha-1 Antitrypsin Deficiency (AATD)
GlobeNewswire News Room· 2025-08-05 11:00
Core Insights - Beam Therapeutics has made significant progress in its clinical trials, particularly with BEAM-101 for sickle cell disease and BEAM-302 for alpha-1 antitrypsin deficiency, with key data updates expected by early 2026 [2][11][7] Financial Performance - As of June 30, 2025, the company reported cash, cash equivalents, and marketable securities totaling $1.2 billion, an increase from $850.7 million at the end of 2024, providing a cash runway expected to support operations into 2028 [10][11] - Research and Development (R&D) expenses for Q2 2025 were $101.8 million, up from $87.0 million in Q2 2024, while General & Administrative (G&A) expenses decreased to $26.9 million from $29.6 million in the same period [11][16] - The net loss for Q2 2025 was $102.3 million, or $1.00 per share, compared to a net loss of $91.1 million, or $1.11 per share, in Q2 2024 [11][16] Clinical Development Progress - The BEACON Phase 1/2 trial of BEAM-101 has completed dosing for 30 patients, including the first adolescent patient, with plans for further data presentation at the end of 2025 [11][2] - The BEAM-302 trial has dosed 17 patients, showing promising results in restoring alpha-1 antitrypsin levels and reducing disease-causing mutations, with a full program update expected in early 2026 [3][5][7] - The company is expanding the dose exploration phase of BEAM-302 and has initiated enrollment in Part B of the trial, targeting patients with mild to moderate liver disease [4][5] Regulatory Designations - BEAM-101 received orphan drug designation from the FDA, aimed at supporting the development of treatments for rare diseases [11] - BEAM-302 was granted both Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designations by the FDA, facilitating its development for serious unmet medical needs [11]
MeiraGTx Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for AAV-GAD for the Treatment of Parkinson’s Disease
Globenewswire· 2025-05-09 12:00
Core Viewpoint - The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to MeiraGTx's AAV-GAD for treating Parkinson's disease, indicating significant potential benefits based on clinical study data [1][2][3] Company Overview - MeiraGTx Holdings plc is a clinical-stage genetic medicines company with a focus on developing gene therapies, including AAV-GAD for Parkinson's disease [1][7] - The company has a broad pipeline with four late-stage clinical programs targeting various diseases, including inherited conditions and common diseases [7] Product Details - AAV-GAD is an investigational gene therapy designed to address the dysfunction of brain circuits in Parkinson's disease by promoting local production of GABA [6] - The therapy is administered via a one-time stereotactic infusion into the subthalamic nucleus, a critical area for movement regulation [6] Clinical Studies - The RMAT designation was awarded following positive results from three clinical studies: a Phase 1 study (n=12), a Phase 2 double-blind study (n=45), and a bridging study (n=14) [2][3] - The Phase 2 studies demonstrated significant benefits on the standard motor endpoint in Parkinson's disease, specifically in the UPDRS Part 3 [3] Regulatory Insights - RMAT designation allows for increased interaction with the FDA, facilitating expedited development and review processes for promising therapies [4] - The designation includes benefits similar to Fast Track and Breakthrough Therapy designations, such as rolling review and potential Priority Review for the biologics license application [4] Market Context - Parkinson's disease is the second most common neurodegenerative disease, affecting nearly one million people in the U.S. and over 10 million globally [5] - Many patients experience diminishing returns from traditional dopamine replacement therapies, highlighting the need for effective disease-modifying treatments [5]
enGene Holdings (ENGN) 2025 Conference Transcript
2025-05-05 18:00
Summary of enGene Holdings (ENGN) Conference Call Company Overview - **Company Name**: enGene Holdings (ENGN) - **Headquarters**: Montreal, Canada; Corporate Offices in Boston, USA - **Industry**: Biotechnology, specifically focused on genetic medicines - **Ticker Symbol**: Listed on NASDAQ Core Points and Arguments 1. **Market Opportunity**: The company is developing ditalimogene voraplasmin for non-muscle invasive bladder cancer (NMIBC), a market expected to grow significantly due to the introduction of new agents, similar to the multiple myeloma market which expanded from $1 billion to over $20 billion with new treatments [6][5][4] 2. **Clinical Development Timeline**: Enrollment for the pivotal study will be completed this year, with top-line data expected next year and a BLA filing anticipated for 2027 [7][9] 3. **Product Advantages**: Ditalimogene voraplasmin is a genetic medicine that has shown good efficacy (71% complete response rate) and excellent tolerability, with a unique formulation that allows for easy handling and storage [8][18][32] 4. **Cash Position**: The company has a strong cash position of approximately $275 million, which is expected to last until 2027, providing a buffer against financial risks [9] 5. **Unmet Medical Need**: NMIBC has a significant unmet medical need, with current treatments like BCG in short supply and associated with severe side effects [15][16][18] 6. **Regulatory Clarity**: The company believes it has a clear regulatory path, with existing clinical data and a well-characterized manufacturing process, which is crucial for biotech success [50][51] Additional Important Content 1. **Patient Demographics**: NMIBC predominantly affects older males, with an average age in their mid-seventies, and about 730,000 individuals in the U.S. are affected [11][30] 2. **Treatment Landscape**: Current treatments are limited, with BCG being the first-line therapy but facing supply issues. Other approved agents have limitations in efficacy and adverse events [16][17][18] 3. **Manufacturing Advantages**: enGene has already established a scalable manufacturing process, which is expected to provide a commercial advantage due to lower costs [21][24] 4. **Development Program**: The Legend program includes multiple cohorts, targeting BCG-unresponsive patients and exploring other applications for the platform [25][26] 5. **Efficacy Comparison**: The company anticipates that protocol changes will improve efficacy rates, which are currently competitive with existing treatments [39][40][41] 6. **Team Experience**: The management team has extensive experience in biotech and pharmaceuticals, enhancing the company's credibility and operational capability [49] This summary encapsulates the key points discussed during the enGene Holdings conference call, highlighting the company's strategic positioning within the biotechnology sector and its focus on addressing significant medical needs in bladder cancer treatment.