FDA Clears First Trial of Prime Medicine’s Editing Therapy & More — This Week in RARE Daily

May 3, 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 26-May 2, 2024:

FDA Clears First Trial of Prime Medicine’s Editing Therapy
The FDA has granted clearance to Prime Medicine to begin human clinical trials of an experimental prime editing therapy for the rare, autoimmune condition chronic granulomatous disease, the first in a new class of one-time curative genetic therapies.
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ONO to Acquire Deciphera for $2.4 Billion
ONO Pharmaceutical agreed to acquire Deciphera Pharmaceuticals, a company with a portfolio of therapeutics for rare cancers, for $2.4 billion.

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FDA Approves X4 Pharma’s Xolremdi for WHIM Syndrome
The FDA has approved X4 Pharmaceuticals’ Xolremdi capsules for use in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes.
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TeleRare Health Launches National Virtual Clinic for People with Rare Diseases
TeleRare Health has launched service to provide virtual care for people and their families with rare and genomic diseases nationwide. Patients will be able to get genetic testing, and ultimately ensure patients benefit from the AI revolution in genomics.

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AI Used to Identify Undiagnosed Patients with Immune Disorder through EHRs
Researchers used artificial intelligence to to review electronic health records and identify people suspected of having an undiagnosed, rare immunodeficency.

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