Clinical Pipeline and Regulatory Milestones - The company has 3 late-stage clinical programs in pivotal/Phase 3 trials for prevalent non-inherited indications[4,7,30,36,42,90] - Potential global filings are anticipated in 2025, 2026, and 2027[7,30,36,42,90] - The company has a deep pre-IND pipeline targeting conditions like ALS, MC4R obesity, and metabolic disease[7,8,32,38,45,93] Manufacturing and Technology - The company operates 2 GMP facilities at commercial scale[5,9,33,39,46,94] - Proprietary vectorization technology increases potency by 2-10x from the same promoter[5,9,34,40,47,95] - AI-driven improvements are based on over 20 vectors and more than 50 GMP runs[5,9,33,39,46,94] Partnerships and Financials - MeiraGTx will receive up to $415 million from Janssen through an asset purchase agreement[11] - Sanofi made a $30 million strategic investment through the sale of 4 million ordinary shares at $7.50 per share[11] AAV-AQP1 for Radiation Induced Xerostomia - There are 170,000 Grade 2/3 RIX patients in the US[13] - There are 15,000 new cases of grade 2/3 RIX annually in the US[13] AAV-GAD for Parkinson's Disease - There are 10 million Parkinson's patients worldwide[18,28] - The estimated economic burden of Parkinson's Disease in the US is $52 billion[19]

Up to $40 Million Private Equity Financing into a New Entity Addressing Cardiac Genetic Diseases that Existing AAV Platforms are Unable to Treat Lexeo Contributing Expertise and Know-How in Cardiac Genetic Medicines, Preclinical Intellectual Property, and Technology, in Combination with Novel Non-Viral RNA Delivery Platform Represents Pipeline Diversification and Advancement of Leading-Edge Cardiovascular Science NEW YORK, June 24, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical ...

Core Viewpoint - Krystal Biotech, Inc. has initiated its Phase 3 clinical trial named "IOLITE" to evaluate KB803 for treating and preventing corneal abrasions in patients with dystrophic epidermolysis bullosa (DEB) [1][2] Group 1: Clinical Trial Details - IOLITE is a double-blind, multicenter, placebo-controlled study with a crossover design, focusing on KB803 administered as eye drops [4] - The trial will enroll approximately 16 subjects, with a primary endpoint measuring the change in average days per month with corneal abrasion symptoms while receiving KB803 versus placebo [5][6] - Patients must complete a 12-week run-in period in an ongoing natural history study before participating in IOLITE [4][5] Group 2: Patient Population and Disease Impact - Over 25% of DEB patients are believed to experience ocular complications, with more than 50% of patients suffering from the recessive form of DEB (RDEB) [2] - The estimated number of DEB patients in the U.S. exceeds 750, with over 2,000 affected globally [2] - Current management of DEB-related ocular complications is limited to supportive care and surgical interventions, with no corrective therapies available [2] Group 3: Previous Findings and Compassionate Use - A previous compassionate use case of KB803 demonstrated full corneal healing at three months and significant visual acuity improvement from hand motion to 20/25 by eight months [1][12] - The compassionate use case involved a patient with severe cicatrizing conjunctivitis secondary to DEB, treated with regular B-VEC administration [11][12] Group 4: Company Overview - Krystal Biotech, Inc. is a global biotechnology company focused on developing genetic medicines for diseases with high unmet medical needs [13] - The company’s first commercial product, VYJUVEK®, is the first-ever redosable gene therapy approved by the FDA and EMA for treating dystrophic epidermolysis bullosa [13]

Solid Biosciences (SLDB) 2025 Conference June 04, 2025 02:00 PM ET Speaker0 Everyone. My name is Maury Raycroft and I'm one of the biotech analysts at Jefferies. It's with great pleasure that I'd like to welcome the SolidBio team. We've got Bo Kumbow and Gabe Brooks joining us today. And we're going to do fireside chat format. So, for those who are new to the story, maybe give a brief intro to Solid, your key programs and platform. Speaker1 Yeah, thank you, Mari. I appreciate the invitation. Solid Bioscienc ...

Company Participation - Krystal Biotech, Inc. will participate in the Goldman Sachs 46th Annual Global Healthcare Conference on June 10, 2025, in Miami [1] - Krish S. Krishnan, Chairman and CEO, will engage in a fireside chat at 2:00 pm ET and host investor meetings throughout the day [1] Webcast Information - A webcast of the presentation will be available starting at 2:00 pm ET on June 10, 2025, and will be posted on the Investors section of 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 by the FDA and EMA for treating 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]

Solid Biosciences Investor Contact: CHARLESTOWN, Mass., May 28, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company" or "Solid"), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, announced that Bo Cumbo, President and CEO, and Gabriel Brooks, M.D., Chief Medical Officer, will participate in a fireside chat at the Jefferies Global Healthcare Conference on Wednesday, June 4, at 2:00 pm ET. A live webcast of the fireside chat will b ...

Core Viewpoint - Lexeo Therapeutics has successfully secured approximately $80 million through a private placement of common stock and pre-funded warrants, extending its cash runway into 2028 to support ongoing clinical programs and operations [1][4]. Group 1: Financing Details - The private placement involves the issuance of 20,790,120 shares of common stock and pre-funded warrants for 6,963,556 shares, with a purchase price of $2.8825 per share [1]. - The financing was co-led by Frazier Life Sciences and Janus Henderson Investors, with participation from various new and existing investors [2]. - The transaction is expected to close on May 28, 2025, subject to customary closing conditions [1]. Group 2: Use of Proceeds - Lexeo plans to utilize the net proceeds from the private placement to advance its clinical stage programs, as well as for working capital and general corporate purposes [4]. - The combined proceeds from this placement and existing cash are projected to fund operations and capital expenditures into 2028 [4]. Group 3: Company Strategy and Leadership - The CEO of Lexeo Therapeutics emphasized that this financing will enhance the company's leadership in cardiac genetic medicines and support the acceleration of their clinical pipeline [3]. - Lexeo is focused on developing next-generation therapies targeting the genetic causes of cardiovascular diseases, including LX2006 for Friedreich ataxia cardiomyopathy [8].

CAMBRIDGE, Mass., May 12, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor designed to correct the disease-causing mutation in patients with al ...

- This RMAT designation is based on data from 3 clinical studies demonstrating the potential benefit of AAV-GAD as a one-time treatment for Parkinson’s disease - RMAT designation includes the benefits of the Fast Track and Breakthrough Therapy designations, allows frequent regulatory interactions with the FDA, and potential routes to accelerated approval and Priority Review LONDON and NEW YORK, May 09, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage gen ...

enGene Holdings (ENGN) 2025 Conference May 05, 2025 02:00 PM ET Speaker0 For our next presentation, it's from NGIN, and we have the pleasure of having Ron Cooper, the CEO, here to present for us. So take it away, Ron. Thank you. Speaker1 Well, good afternoon, everybody. It's a real pleasure to be here. You know, first of all, thanks to the conference organizers. It's great to be back in back in Canada. NGEN is a company form formed in Canada. Our headquarters is in Montreal. Our labs are there, and we have ...