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The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

Worldwide Clinical Trials

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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Vertex gearing up for launch as sickle cell therapy review advances

Bio Pharma Dive

Executives at the biotech say they’re trying to get ahead of the payer and production challenges that will face their gene editing treatment exa-cel, which is now under FDA review.

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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. The company will also receive tiered royalties on the worldwide net sales of products developed from the collaboration.

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Asklepios partners with ReCode on gene-editing platform

Pharmaceutical Technology

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

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ElevateBio raises funds to advance cell and gene therapies

Pharmaceutical Technology

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. Life Edit will receive an upfront cash payment and $335m in potential development, regulatory and commercial milestones for each of the first two programmes.

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Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Worldwide Clinical Trials

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. There are also nuances for operational planning in cell therapy development programs.

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Verve Therapeutics and Lilly partner to advance gene editing programme

Pharmaceutical Technology

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). The company will receive a combined upfront payment and equity investment of $60m.