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FDA Action Alert: Vertex/CRISPR, Arcutis and Optinose

BioSpace

This week, the FDA could approve the first CRISPR-edited therapy in the U.S., while two other companies await decisions on topical drugs.

Drugs 115
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Vertex signs licence deal with CRISPR Therapeutics for diabetes therapies

Pharmaceutical Technology

Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). It originated under the partnership of CRISPR Therapeutics and ViaCyte. Topic sponsors are not involved in the creation of editorial content.

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Editas grabs orphan drug status for sickle cell disease CRISPR therapy

Pharmaceutical Technology

EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. Topic sponsors are not involved in the creation of editorial content.

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Scribe and Sanofi partner to develop cell therapies for cancer

Pharmaceutical Technology

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Topic sponsors are not involved in the creation of editorial content.

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BrightPath and Artisan Bio sign agreement to expedite cell therapies

Pharmaceutical Technology

BrightPath will obtain non-exclusive rights to the STAR-CRISPR editing platform of Artisan Bio for accelerating the development of BrightPath’s iNKT cells. Topic sponsors are not involved in the creation of editorial content. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Vertex signs agreement to acquire ViaCyte for $320m

Pharmaceutical Technology

Furthermore, through the takeover, Vertex will obtain access to new hypoimmune stem cell assets under the ViaCyte-CRISPR Therapeutics partnership. Topic sponsors are not involved in the creation of editorial content. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Bluebird moves forward in sickle cell arena with lovo-cel BLA

Pharmaceutical Technology

The BLA filing brings bluebird closer to its competitors CRISPR Therapeutics and Vertex Pharmaceuticals , which finished a rolling BLA for their CRISPR-based gene therapy exa-cel for the same condition earlier this month. Topic sponsors are not involved in the creation of editorial content.