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Magazine: CRISPR gene therapies cut through in 2023

Pharmaceutical Technology

If things go as per plan, in a few months, the US Food and Drug Administration (FDA) will deliberate on the first-of-its-kind CRISPR-based gene therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The post Magazine: CRISPR gene therapies cut through in 2023 appeared first on Pharmaceutical Technology.

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Sanofi invests $30m in MeiraGTx’s gene therapy pipeline

BioPharma Reporter

The London and New York-based gene therapy player MeiraGTx Holdings has received a $30 million investment from Sanofi in addition to potential strategic deals with the big pharma company down the road.

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Novartis adds gene therapy for blindness with Gyroscope buy

pharmaphorum

Gyroscope Therapeutics and its gene therapy for the sight-robbing disease geographic atrophy (GA) has been acquired by a big pharma company – but likely not the one you would expect. CFI regulates the activity of the complement immune system which is over-activated in GA. It has been fast-tracked by the FDA.

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Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective 

Pharmaceutical Technology

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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The Unprecedented Growth of Cell and Gene Therapy (2021)

Cloudbyz

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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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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Italfarmaco’s Duvyzat Wins FDA Approval as First Nonsteroidal Treatment for All Genetic Variants of DMD

XTalks

Bettica said the company’s focus is to now “make Duvyzat available as a treatment for DMD management in the US as quickly as possible.” In November, the FDA extended its decision deadline by three months to allow extra time for regulators to review additional information submitted by Italfarmaco as part of a new drug application.