Malicious jumping genes meet their match: Study reveals cell's adaptive piRNA defenses
Adverse genetic mutations can cause harm and are due to various circumstances. "Jumping genes" are one cause of mutations, ...
AI helps find promising therapeutic target for rare salivary gland cancer
Researchers have uncovered a promising therapeutic target for adenoid cystic carcinoma (ACC), a rare cancer of the salivary ...
The American Journal of Managed Care2h
Dr Toon Van Gorp: Mirvetuximab Soravtansine Should Be Global Standard for FRα+ Ovarian Cancer
Toon Van Gorp, MD, PhD, a MIRASOL trial investigator, emphasizes that the final analysis reinforces the efficacy of mirvetuximab soravtansine-gynx (Elahere; AbbVie) in patients with folate receptor ...
Breakthrough gene therapy alleviates Dravet syndrome symptoms in mice without side effects
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
The American Journal of Managed Care1d
EMBARK Data Show Continued Improvements With DMD Gene Therapy
Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...
STAT1d
Novartis finally delivers gene therapy data in older SMA patients. Will they still want it?
In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
Can-Fite to Initiate Phase II Study in the Rare Genetic Disease Lowe Syndrome with Piclidenoson
FDA & EMA approvals for rare genetic diseases are fast and require clinical studies with small number of patients Ramat Gan, Israel, March ...
STAT1d
Prime and Beam, born of same lab, pursuing rival gene-editing treatments
Prime Medicine and Beam Therapeutics had common origins in gene editing. Now they're both pursuing treatments for the same ...
The Pharma Letter5d
Regenxbio completes its BLA filing for first potential gene therapy for MPS II
US biotech Regenxbio (Nasdaq: RGNX) has successfully completed the BLA submission for its mucopolysaccharidosis II (MPS II) ...
The Prototype: NASA Closes Chief Scientist’s Office
Plus: Gene therapy with bioengineered blood cells, a possible quantum computing milestone, a new space telescope, how to not ...
REGENXBIO Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Operational Updates
REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and recent operational highlights for the fourth quarter and year ended December 31, 2024.