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.
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.
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.
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.
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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