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FDA Grants Sarepta Expanded Approval for DMD Gene Therapy & More — This Week in RARE Daily

June 21, 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, June 14 – 20, 2024:

FDA Grants Sarepta Expanded Approval for DMD Gene Therapy

The U.S. Food and Drug Administration granted Sarepta Therapeutics an expansion to the labeled indication for its Duchenne muscular dystrophy gene therapy Elevidys to include individuals with a confirmed mutation in the DMD gene who are at least 4 years of age.

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Day One Expands Pipeline with Antibody Drug Conjugate Targeting PTK7

Day One Biopharmaceuticals entered into an exclusive licensing agreement with MabCare Therapeutics for MTX-13, a novel ADC targeting protein-tyrosine kinase 7.

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Roche and Ascidian enter RNA Exon Editing Collaboration for Neurological Diseases

Ascidian Therapeutics entered into a research collaboration and licensing agreement with Roche for the discovery and development of RNA exon editing therapeutics targeting neurological diseases.

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Repurposed Drug May Help Stabilize Vision in Ultra-Rare Disease

A new study from researchers at Washington University School of Medicine in St. Louis found that the rare condition known as retinal vasculopathy with cerebral leukoencephalopathy and systemic manifestations may be stabilized by the use of an FDA-approved drug for a different condition.

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Ovid Tumbles as Takeda’s Soticlestat Misses in Phase 3 Studies in Rare Epilepsies

Takeda said its experimental therapy soticlestat narrowly missed it primary endpoint of a reduction in convulsive seizure frequency in Dravet syndrome while showing clinically meaningful and nominally significant effects in multiple key secondary efficacy endpoints.

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