Profluent Releases AI-Created and Open-Source Gene Editor & More — This Week in RARE Daily

April 25, 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 19-25, 2024:

Profluent Releases AI-Created and Open-Source Gene Editor
Profluent said it demonstrated the first successful precision editing of the human genome with a customizable gene editor designed from scratch with AI as it unveiled its OpenCRISPR initiative to make the gene editor freely available to license for ethical research and commercial uses.

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LifeArc Invests $50 Million to Launch Rare Disease Research Centers
UK charity LifeArc is investing $50 million (£40 million) to launch four new LifeArc Translational Centres for Rare Diseases to provide people living with rare medical conditions access to improved tests, treatments, and potentially cures.

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FDA Grants Accelerated Approval to Day One’s Ojemda for Childhood Brain Tumor
The FDA has granted accelerated approval to Day One Biopharmaceuticals’ Ojemda for a type of rare, childhood brain tumor for patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.

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Rett Syndrome Research Trust Partners with Vivalink to Accelerate Research
Digital health technology provider Vivalink is collaborating with the Rett Syndrome Research Trust (RSRT) to advance research into the rare, neurological disorder. Vivalink will provide its Multi-Vital ECG wearable technology and data services for heart rate and oxygen saturation monitoring to support the RSRT’s new VIBRANT study.

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Regeneron and Mammoth Collaborate on Next-Gen CRISPR Gene Editing for Multiple Diseases
Regeneron and Mammoth will collaborate to research, develop and commercialize in vivo CRISPR-based gene editing therapies for multiple tissues and cell types by leveraging Regeneron’s expertise in AAV and antibody engineering and Mammoth’s expertise in ultracompact gene editing systems.

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