NCATS Seeks Applicants for Rare Disease Clinical Research Funding & More — This Week in RARE Daily

April 5, 2024

This Week in RARE Daily is a feature from Global Genes where you can get a quick rundown of the top 5 headlines in the rare disease space from our editorial staff. Here are top stories from this past week, March 29-April 4, 2024:

NCATS Seeks Applicants for Rare Disease Clinical Research Consortia Funding
The NIH’s National Center for Advancing Translational Sciences is seeking applicants for its Rare Diseases Clinical Research Consortia, which is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment, and data sharing.

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Amylyx to Remove ALS Drug from Market and Cut 70 Percent of Workforce
Amylyx Pharmaceuticals is Relyvrio from the U.S and Canadian markets and cutting up to 70 percent of its workforce in a corporate restructuring to focus on upcoming clinical milestones and extend its cash runway into 2026.

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Ring Publishes Study of Novel Gene Delivery Platform that Could Enable Redosable Gene Therapies
Ring Therapeutics has debuted the first gene delivery vector system based on a human commensal virus enabling potentially redosable, programmable viral vectors.

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CF Foundation Invests Up to $8.5 Million in SpliSense
Cystic Fibrosis Foundation is investing additional funds in SpliSense to continue clinical trials for its inhaled antisense oligonucleotide therapy for people with rare pulmonary disease cystic fibrosis who have certain splicing and potentially other rare mutations.

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Praxis Raises $200 Million in Public Offering
Praxis Precision Medicines raised $200 million through a public offering to advance its clinical stage genetic medicines pipeline for central nervous system disorders characterized by neuronal excitation-inhibition imbalance.

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