Core Insights - Sarepta Therapeutics is advancing its investigational siRNA treatment SRP-1003 for Myotonic Dystrophy Type 1, with positive progress reported in the Phase 1/2 clinical study [1][2] Study Progress - Cohorts 1 (1.5 mg/kg) and 2 (3 mg/kg) of the study are complete, while cohort 3 (4.5 mg/kg) is fully enrolled and ongoing [2] - Patients are currently being dosed in cohort 4 (6 mg/kg), with plans to initiate dosing in cohort 5 (12 mg/kg) in early 2026 [3] Financial Milestones - Following a positive review by the drug safety committee, a second milestone payment of USD 200 million will be made to Arrowhead Pharmaceuticals within 60 days [2] siRNA Platform Overview - Sarepta's siRNA platform focuses on therapies for neurodegenerative and pulmonary diseases, with ongoing preclinical programs for Spinocerebellar ataxia types 1 and 3 [3] Company Mission - Sarepta is dedicated to engineering precision genetic medicine for rare diseases, holding leadership positions in Duchenne muscular dystrophy and expanding its portfolio across various disease areas [4]

SAN DIEGO, Nov. 17, 2025 (GLOBE NEWSWIRE) -- Calidi Biotherapeutics, Inc. (“Calidi” or the “Company”) (NYSE American: CLDI), a clinical-stage biotechnology company pioneering the development of targeted therapies with the potential to deliver genetic medicines to distal sites of disease, is pleased to invite investors to a webinar on November 19, 2025, at 1:15 p.m. PT. The exclusive event, hosted by RedChip Companies, will feature Calidi’s CEO, Eric Poma, Ph.D. Attendees will gain insights into Calidi’s pio ...

Company Overview - Solid Biosciences is a precision genetic medicine company focusing on gene therapy [2] - The company has three main drugs in the clinic, with IND open and patient dosing expected this year [2] - The targeted conditions for these drugs include Duchenne muscular dystrophy, Friedreich Ataxia, and catecholaminergic polymorphic ventricular tachycardia (CPVT) [2] - A new program for dilated cardiomyopathy targeting TNNT2 is anticipated to launch next year [2] - The company emphasizes advancements in next-generation delivery systems, including capsids, promoters, and manufacturing processes [2] - Solid Biosciences is based in the Boston area and employs approximately 110 people [2]

Core Insights - Calidi Biotherapeutics is hosting a webinar on July 24, 2025, to discuss its innovative approach to precision genetic medicine using its proprietary RedTail™ platform [1][2] - The RedTail platform is designed to deliver genetic therapies to difficult-to-treat tumors while evading immune detection, showcasing promising preclinical data [2][5] - The oncology market is projected to exceed $560 billion by 2033, presenting significant growth opportunities for Calidi [2] Company Overview - Calidi Biotherapeutics is a clinical-stage biotechnology company focused on developing targeted therapies for delivering genetic medicines to disease sites [5][6] - The RedTail platform has been developed over a decade and utilizes engineered viral vectors to effectively reach tumor sites and deliver gene therapies [5][6] - The lead candidate, CLD-401, is currently in IND-enabling studies and targets non-small cell lung cancer, ovarian cancer, and other high unmet medical need tumor types [6] Strategic Initiatives - The company is advancing towards IND filing and exploring strategic partnerships with pharmaceutical companies for global commercialization [2] - Calidi's business model is capital-efficient, with a strong balance sheet and a next-generation pipeline targeting both cancer and autoimmune diseases [2]

Core Insights - Solid Biosciences Inc. is participating in the Jefferies Global Healthcare Conference on June 4, 2025, with a fireside chat featuring the President and CEO, Bo Cumbo, and Chief Medical Officer, Gabriel Brooks [1][2] Company Overview - Solid Biosciences is focused on developing precision genetic medicines targeting rare neuromuscular and cardiac diseases, including Duchenne muscular dystrophy, Friedreich's ataxia, and catecholaminergic polymorphic ventricular tachycardia [3] - The company is advancing a diverse pipeline of gene therapy candidates and innovative libraries of genetic regulators aimed at improving gene therapy delivery across the industry [3] - Solid Biosciences was founded by individuals directly impacted by Duchenne, emphasizing its mission to enhance the daily lives of patients with rare diseases [3]

Core Insights - Solid Biosciences Inc. is participating in The Citizens Life Sciences Conference on May 7, 2025, with CEO Bo Cumbo leading a fireside chat [1][2] Company Overview - Solid Biosciences is focused on developing precision genetic medicines targeting rare neuromuscular and cardiac diseases, including Duchenne muscular dystrophy, Friedreich's ataxia, and catecholaminergic polymorphic ventricular tachycardia [3] - The company is advancing a diverse pipeline of gene therapy candidates and innovative technologies aimed at improving gene therapy delivery across the industry [3] - Solid Biosciences was founded by individuals directly impacted by Duchenne, emphasizing its mission to enhance the daily lives of patients with rare diseases [3]