Announcements
Excerpt from the Press Release: LA JOLLA, Calif.–(BUSINESS WIRE)–Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced positive topline data from the PACIFIC Study evaluating bexicaserin (LP352), a potentially best-in-class and highly selective, oral, novel 5-HT2C receptor superagonist for seizures associated with a broad…
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– In Phase 1/2 ACHIEVE Trial, DYNE-101 Demonstrated Dose-Dependent Splicing Correction, Muscle Delivery and DMPK Knockdown – – All Evaluable Patients in the 3.4 mg/kg Cohort Treated with DYNE-101 Q4W Demonstrated Consistent Splicing Correction with a 19% Mean Improvement Across 22-Gene Panel at 3 Months – – Improvement in Myotonia (vHOT) as well as Fatigue…
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Kara N. Fitzgerald1 , Tish Campbell2 , Suzanne Makarem2 , Romilly Hodges3 Excerpt from the Press Release: Abstract Here we report on a case series of six women who completed a methylation-supportive diet and lifestyle program designed to impact DNA methylation and measures of biological aging. The intervention consisted of an 8-week program that included…
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Clinical Success Achieved in Second Cohort of Patients Excerpt from the Press Release: PRINCETON, N.J., Jan. 4, 2024 /PRNewswire/ — Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today preliminary top-line results of its…
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Excerpt from the Press Release: SAN FRANCISCO AND BOSTON, Jan. 3, 2024 /PRNewswire/ — MapLight Therapeutics, a clinical-stage biopharmaceutical company working to develop targeted novel therapeutics to improve the lives of people with brain disorders, today announced completion of a Phase 1 clinical trial evaluating the bioavailability, safety, and tolerability of an extended release formulation…
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Phase 1 study aims to evaluate safety and tolerability of a single ascending dose of RCT1100 in patients with PCD caused by mutations in the DNAI1 gene RCT1100 is the first clinical evaluation of an inhaled mRNA-based therapy designed to restore ciliary function in people with PCD Excerpt from the Press Release: MENLO PARK, Calif.–(BUSINESS…
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B cells are prolific producers of antibodies, but for the first time, scientists have modified them to make other proteins to counteract a serious genetic disease. Excerpt from the Press Release: Our B cells help prevent us from getting sick. Their job is to make antibodies, immune system proteins that fight off viruses and other…
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Excerpt from the Press Release: CAMBRIDGE, Mass., Dec. 14, 2023 /PRNewswire/ — NeuroSense Therapeutics Ltd. (Nasdaq: NRSN) (“NeuroSense”), a company developing treatments for severe neurodegenerative diseases, today reported additional positive efficacy data from further evaluation of its Phase 2b trial (PARADIGM) with NeuroSense’s lead drug candidate for amyotrophic lateral sclerosis (“ALS”), PrimeC. Evaluation of the pre-specified…
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Excerpt from the Press Release: CAMBRIDGE, Mass., Dec. 14, 2023 (GLOBE NEWSWIRE) — Cullinan Oncology, Inc. (Nasdaq: CGEM), a biopharmaceutical company focused on modality-agnostic, targeted oncology therapies, today announced that the first patient has been dosed with CLN-617 in a Phase 1 clinical trial. This is a first-in-human, multi-center, open label study designed to evaluate the…
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Maze Therapeutics Announces Initiation of Dosing in Phase 1 Trial Evaluating MZE829, a Novel Oral APOL1 Inhibitor, as a Potential Treatment for APOL1 Kidney Disease Excerpt from the Press Release: SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Maze Therapeutics, a company translating genetic insights into new precision medicines, today announced the initiation of dosing in the company’s Phase…
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