CRISPR Therapeutics
NEWS
With this milestone, the field is now wide open for the next in vivo CRISPR success, and Editas is hoping that it will be in the ocular disease space.
The company is eager to diversify, putting Phase II clinical trials in sickle-cell disease, beta-thalassemia, kidney diseases, and pain management.
Jennifer Doudna is best known for discovering CRISPR/Cas9 gene editing with Emmanuelle Charpentier, and both winning the Nobel Prize in Chemistry in 2020.
Interim results from the Phase I study presented at the Peripheral Nerve Society Annual Meeting demonstrated that the investigational therapy, NTLA-2001, greatly reduced the disease-causing protein after a single infusion.
Per the agreement, CRISPR will lead the research and development of the Friedreich’s ataxia program, while Capsida will shepherd the R&D for the ALS effort.
Under the terms of the expanded deal, Vertex will be responsible for 60% of the costs of developing, manufacturing and commercializing CTX001 with support from CRISPR Therapeutics, and will receive 60% of profits from global sales.
Biopharma and life sciences companies strengthen their leadership teams and boards with these Movers & Shakers.
It was another busy week for clinical trial updates and news. Here’s a look.
Despite promising Phase I data for its CRISPR-based allogeneic CAR-T therapy, CRISPR Therapeutics may have a safety issue to contend with.
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