Neurogene: Interesting CNS Disease Company, Data Coming Soon
Neurogene has a $903M market cap with two phase 1/2 assets, NGN-401 for Rett Syndrome and NGN-101 for CLN5 Batten Disease.
Frontiers1d
Innovative Malaria Control Strategies: Success Stories from Diverse Global Regions
Malaria remains a significant global health challenge, with approximately 249 million cases reported annually. Despite being ...
precisionmedicineonline1d
Rocket Pharma Starts Rolling BLA Submission for Fanconi Anemia Gene Therapy
RP-L102, which the firm designed to treat a form of disease caused by FANCA mutations, is already under review with the European Medicines Agency.
The American Journal of Managed Care3d
Fidanacogene Elaparvovec Shows Strong Results in Open-Label Phase 3 Study
The gene therapy improved annualized bleed rates, though a handful of study participants resumed factor IX prophylaxis.
optometryadvisor3d
Novel Gene Therapy Shows Promise for Treating Diabetic Retinopathy
ABBV-RGX-314, administered once within an office setting, can significantly improve disease severity in patients with NPDR.
Stem cells can tailor their role in gene therapy based on the underlying disease, study suggests
RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic ...
Morningstar4d
Positive Long-Term Outlook for Crispr Driven by Casgevy and Diverse Gene Editing Portfolio
LentiGlobin (marketed under the name Lyfgenia) uses the same BB305 viral vector as Zynteglo to deliver a working gene to a patient’s blood cells. In July 2021, the European Medicines Agency’s safety ...
Advances in taro research: New gene silencing system enables rapid gene function verification
A research team has established a virus-induced gene silencing (VIGS) system in taro, enabling researchers to rapidly verify ...
University of Pennsylvania gene therapy spinout Interius BioTherapeutics doses patient, achieves CAR therapy first
Its lead gene therapy candidate has the potential to be faster, less expensive, and more widely available than current CAR T ...