Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases.
Gene therapy pioneer Jim Wilson and researcher Kiran Musunuru are using contrasting approaches to overcome “irrational ...
A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as Zolgensma, could be approved for a broad range of spinal muscular atrophy ...
Researchers have uncovered a promising therapeutic target for adenoid cystic carcinoma (ACC), a rare cancer of the salivary ...
Indian vaccine maker Bharat Biotech said on Thursday it had invested $75 million in its first cell and gene therapy (CGT) facility in the southern Indian state of Telangana and expects its new ...
In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
The future of medicine is being shaped by a confluence of factors at play. If there are worries galore around drug resistant ...
Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...
Pharmaceutical Technology on MSN6d
MeiraGTx and Hologen launch AI-backed gene therapy ventureThe venture aims to advance MeiraGTx’s Parkinson’s gene therapy through Phase III trials with up to $430m in funding from ...
The joint venture with generative AI firm Hologen hands MeiraGTx $200 million up front as well as other financial perks — a “transformative” deal, according to the company’s CEO.
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback