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Ginkgo Bioworks partners with WARF for new GD2 CAR T-cell therapies

Pharmaceutical Technology

The company can use its mammalian cell engineering expertise and capabilities to screen CAR T-cells, to discover and optimise next-generation therapeutic candidates for its partners. All the new GD2 CAR designs developed through the partnership will be validated via in vitro screens and in vivo murine models.

In-Vivo 264
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Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

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Avista and Roche to develop gene therapy vectors for eyes

Pharmaceutical Technology

Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, has entered a collaboration with Roche for developing new AAV gene therapy vectors for eyes. Roche will use these new capsids to carry out preclinical, clinical and marketing activities for gene therapy programmes. Avista is entitled to receive $7.5m

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Editas grabs orphan drug status for sickle cell disease CRISPR therapy

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The first patient had a total haemoglobin level of 16.4 g/dL and 45.4%

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3rd In Vivo Cell Engineering & Gene Editing Summit

pharmaphorum

Achieve Safe & Targeted In Vivo Delivery of Payloads with Clarity in Regulations & Appropriate Models to Advance the Next Frontier of In Vivo Editing from Research to Clinic

In-Vivo 52
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Scribe and Prevail partner for CRISPR-based genetic medicines

Pharmaceutical Technology

Scribe Therapeutics has entered a strategic collaboration with Eli Lilly and Company subsidiary Prevail Therapeutics for accelerating in vivo CRISPR-based therapies to target the causes of serious neurological and neuromuscular diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Genetics 130
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Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia

XTalks

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.