FDA puts REGENXBIO gene therapy trials on hold after brain tumour

A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.
Fierce Biotech

FDA puts clinical hold on Regenxbio gene therapies weeks before approval ruling

A central nervous system (CNS) tumor has prompted the FDA to place clinical holds on two Regenxbio gene therapies, including ...
BioPharma Dive

Regenxbio gene therapy trials suspended by FDA over safety worries

The development of a brain tumor in a study participant led regulators to suspend a Hurler syndrome therapy in early testing ...
STAT

FDA halts gene therapy trials after brain tumor found in patient

The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
Stocktwits on MSN

RGNX shares pare losses as Wall Street dismisses gene therapy clinical hold spurred selloff as an overreaction

Leerink said the premarket selloff in shares underscores the degree of surprise on the news, feeding into the "FDA ...
News Medical on MSN

First topical CRISPR gene therapy corrects disease-causing mutations in human skin

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

Intellia gets FDA nod to resume gene therapy trial after safety pause

Jan 27 (Reuters) - Intellia Therapeutics said on Tuesday that the U.S. drug regulator has lifted a clinical hold on one of ...

Sarepta's Duchenne gene therapy slows disease progression at three years

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...
Fierce Biotech

From gene therapies to … firearms? Italian biotech makes unexpected strategic pivot

When Genenta Science listed on the Nasdaq at the tail end of 2021, the Milan-based biotech was squarely focused on its pipeline of cell-based gene therapies. | Four years after listing on the Nasdaq, ...
GlobalData on MSN

Genespire to take rare disease gene therapy to clinic in 2026

GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.
Yicai Global

Chinese Scientists Make Key Advance in Using Gene Therapy

(Yicai) Jan. 28 -- Chinese medical researchers have made a breakthrough in gene therapy that overcomes the limitations of ...
Medical Marketing & Media

Medicaid tries new approach with sickle cell: Companies get paid only if costly gene therapies work

Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...