AADC deficiency is a rare and fatal genetic disorder that typically causes severe disability starting during ... Orsini's ...

Regenxbio, a prolific developer of gene therapy technology, will hand Japan’s Nippon Shinyaku certain rights to two ...

Berlin, Germany, and Research Triangle Park, N.C., USA, Jan. 14, 2025 (GLOBE NEWSWIRE) -- Not intended for UK Media Enrollment ongoing in the United States for REGENERATE-PD clinical trial of AB-1005 ...

It’s a kind of control that no one has ever tried before in patients,” Tune’s chief scientific officer said Monday.

Rockville’s RegenxBio has inked a hefty agreement with a Japanese drug company to get two of its treatment candidates to ...

Autism spectrum disorder is a neurological and developmental condition marked by disruptions in brain-signaling that cause ...

How can we ensure that life-saving drugs or genetic therapies reach their intended target cells without causing harmful side effects? Researchers have taken an important step to answer this question.

The US company has said it is launching the Phase III trials in the first half of this year after Phase I talks with the US ...

Researchers have discovered a three-drug combination that enhances the generation of CAR-T cells, a type of cancer immunotherapy, and could improve CAR-T cell production for clinical treatments. The ...

Cancer is a disease of the genome, according to Dr. Elli Papaemmanuil, computational oncologist at MSK. By looking at the ...

Umoja Biopharma has announced the closing of an oversubscribed $100m Series C financing round to progress CAR-T cell therapy pipeline.

Shares of Regenxbio Inc . (NASDAQ: RGNX) surged 18.4% in pre-open trading Tuesday following the announcement of a strategic partnership with Nippon Shinyaku Co., Ltd. for the development and ...