Research & Development

Solid Biosciences’ SGT-212 gains FDA rare paediatric disease status

Solid Biosciences has secured the US Food and Drug Administration (FDA) rare paediatric disease designation for SGT-212, its investigational gene therapy targeting Friedreich’s ataxia (FA).

IASO Bio gains Hong Kong approval for Fucaso application

IASO Biotechnology has received the Hong Kong Department of Health’s approval for its biologics licence application (BLA) for the fully human anti-B-cell maturation antigen (BCMA) chimeric antigen receptor T-cell (CAR-T) therapy, Fucaso (Equecabtagene Autoleucel), for adults with relapsed or refractory multiple myeloma (R/R MM).

BeOne’s sonrotoclax gains priority FDA review for mantle cell lymphoma

The US Food and Drug Administration (FDA) has accepted the new drug application (NDA) for BeOne Medicines’ B-cell lymphoma 2 (BCL2) inhibitor, sonrotoclax, and granted it priority review for use in treating adults with relapsed or refractory mantle cell lymphoma (R/R MCL).

UNICEF and Gavi to widen affordable malaria vaccine access

UNICEF and Gavi, the Global Alliance for Vaccines and Immunisation now known as the Vaccine Alliance, have announced a pricing deal to expand access to the R21/Matrix-M malaria vaccine, aiming to protect almost seven million more children from the disease.

FDA grants accelerated approval for Otsuka’s IgAN therapy

Otsuka Pharmaceutical has secured accelerated approval from the US Food and Drug Administration (FDA) for Voyxact (sibeprenlimab-szsi) to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at disease progression risk.

Novartis secures approval from FDA for Itvisma to treat SMA

Novartis has received approval from the US Food and Drug Administration (FDA) for the Itvisma (onasemnogene abeparvovec-brve) gene replacement therapy to treat spinal muscular atrophy (SMA).

AstraZeneca to expand biomanufacturing in Maryland, US

AstraZeneca has announced a $2bn investment plan to expand its biomanufacturing presence in the US state of Maryland, aiming to support 2,600 jobs at its Frederick and Gaithersburg sites.

Vaximm and BCME link on oral cancer immunotherapy platform

OSR Company Vaximm has signed a non-binding term sheet with Swiss-based life sciences investor BCM Europe (BCME) for a potential exclusive worldwide licence for Vaximm’s oral cancer immunotherapy platform, VXM01.

Aspen closes $115m in Series C to advance Parkinson’s cell therapy

US-based Aspen Neuroscience has closed a Series C funding round, raising $115m to further the clinical development of its lead autologous cell therapy candidate, ANPD001, for moderate-to-advanced Parkinson’s disease.