Financings of Public Rare Disease Drug Developers Soar & More — This Week in RARE Daily

April 11, 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, April 5-11, 2024:

Financings of Public Rare Disease Drug Developers Soar in First Quarter 2024
Companies developing drugs for rare diseases have raised substantial new capital this year, as rare disease focused public equity and debt financings reached $7.1 billion in the first quarter of 2024, up 307 percent from the first quarter of 2023.

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FDA Says It Will Review Stealth’s Application for Barth Syndrome Treatment
After a protracted fight between the agency and company and patient advocates, the FDA is now reviewing Stealth BioTherapeutics’ application for elamipretide which would treat Barth syndrome.

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Amylyx’s Withdrawn ALS Drug Shows Promise in Wolfram Syndrome
Amylyx Pharmaceuticals reported positive interim data from the ongoing phase 2 HELIOS clinical trial of its experimental therapy AMX0035 in adults living with Wolfram syndrome.

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Great Ormond Street Hospital Devises Plan to Expand Access to Gene Therapies
Great Ormond Street Hospital is piloting a new approach to expand access to gene therapies for children living with a rare disease.

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Vertex Agrees to Acquire Alpine Immune Sciences
On the heels of a surge in M&A activity for rare disease assets, Vertex Pharmaceuticals will be acquiring Alpine Immune Sciences, and the acquisition is expected to close later this quarter. Alpine’s portfolio includes a lead experimental therapy povetacicept, a treatment for IgA nephropathy.

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