Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...

Just three months after further scaling back its support for the struggling hemophilia A gene therapy Roctavian, BioMarin is ...

RNA therapeutics target translation rather than DNA, aiming to correct shared protein production errors. By enabling cells to read through premature stop codons, engineered tRNAs could restore ...

CARsgen Therapeutics and Jaguar Gene Therapy had received incentives in 2021 to hire 200 workers each at new facilities.

UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question ...

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Thanks to advances in imaging and diagnostic technologies, clinicians can now detect many genetic disorders in the womb, ...

While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

Intellia is now able to continue both Phase III trials of nex-z. Credit: Piotr Swat / Shutterstock.com (Piotr Swat / Shutterstock.com) The US Food and Drug Administration (FDA) has released the ...

CARsgen Therapeutics and Jaguar Gene Therapy had received incentives in 2021 to hire 200 workers each at new facilities.

Delaying onboarding until FDA approval reduces sunk-cost risk but can impede institutional learning and slow post-approval ...