Amazon Web Services Commits $10 million to Pediatric Rare Disease Research & More — This Week in RARE Daily

June 27, 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 21 – 27, 2024:

Amazon Web Services Commits $10 million to Pediatric Rare Disease Research

Amazon Web Services said it is committing $10 million to empower nonprofit institutions to harness the power of the AWS cloud to advance research for pediatric rare diseases. The initiative will support a growing consortium of hospitals and other institutions that use both cloud computing and artificial intelligence with the goal of accelerating research and discoveries.

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Intellia Reports Positive Clinical Proof-of-Concept for Redosing CRISPR-based Therapy

Intellia Therapeutics presented new data demonstrating, for the first time, the potential for redosing with an experimental in vivo CRISPR/Cas9 genome editing therapy. The data from the ongoing phase 1 study of NTLA-2001, a single-dose treatment in development for transthyretin (ATTR) amyloidosis, were presented at the Peripheral Nerve Society Annual Meeting in Montreal, Canada.

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FDA Approves Argenx Vyvgart Hytrulo for CIDP

The U.S. Food and Drug Administration expanded the indication for Argenx’s Vyvgart Hytrulo to include chronic inflammatory demyelinating polyneuropathy, a rare, autoimmune disorder. Vyvgart Hytrulo is approved for CIDP as a once weekly 30-to-90 second subcutaneous injection. It is the first and only neonatal Fc receptor blocker approved for the treatment of CIDP.

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Taysha Raises $75 Million in Public Offering to Advance Gene Therapy for Rett Syndrome

One week after reporting positive data from phase 1/2 studies of its experimental gene therapy to treat Rett syndrome, Taysha Gene Therapies raised $75 million in an underwritten public offering to advance its pipeline of gene therapies targeting monogenic central nervous system disorders.

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Researchers Discover Hidden DNA Mechanisms of Rare Genetic Diseases

Researchers said they have made a groundbreaking discovery that could significantly advance the understanding of genomic disorders by showing how specific DNA rearrangements called inverted triplications contribute to the development of various genetic diseases.

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