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Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

XTalks

Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.

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Scribe Therapeutics Unveils Fully Integrated Platform for Engineering Breakthrough CRISPR-based Genetic Medicines

BioTech 365

Built by CRISPR leaders including Jennifer Doudna to enable best-in-class in vivo therapeutics that permanently treat the underlying cause of disease Initial $20 million Series A led by Andreessen Horowitz Collaboration with Biogen Inc. focused on development of novel genetic … Continue reading →

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Ginkgo acquires machine learning biotech Patch Biosciences

Drug Discovery World

Ginkgo is also working with Sensible Biotechnologies to develop an in vivo microbial mRNA manufacturing platform. Additionally, Ginkgo announced the successful completion of a manufacturing optimisation program with Biogen, focused on redefining the industry standard for manufacturing recombinant adeno-associated virus (AAV)-based vectors.

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Scribe and Sanofi partner to develop cell therapies for cancer

Pharmaceutical Technology

The alliance will leverage the CRISPR genome editing technologies of Scribe to facilitate in genetic modification of new natural killer (NK) cell therapeutics for cancer. Under the agreement, Sanofi will receive non-exclusive rights to Scribe’s CRISPR by Design platform of wholly-owned enzymes for developing ex vivo NK cell therapies.

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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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Gene editing: beyond the hype

pharmaphorum

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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Leading innovators in transgenic murine models for the pharmaceutical industry

Pharmaceutical Technology

Innovation S-curve for the pharmaceutical industry Transgenic murine models is a key innovation area in pharmaceutical s Transgenic murine models refer to mice that have been genetically altered for the purposes of understanding the in vivo functions of genes.

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