gene therapy
Rare diseases – spinal muscular atrophy
January 30, 2026Medical Communications gene therapy, medical communications, muscle and motor function, muscle weakness, rare diseases, sma
Spinal muscular atrophy (SMA) is a rare disease characterised by voluntary muscle weakness that can lead to difficulties in activities …
Five Facts about spinal muscular atrophy
January 28, 2026Medical Communications Five Facts, Neurology, early diagnosis, five types, gene therapy, muscle weakness, spinal muscular atrophy
1 Spinal muscular atrophy (SMA) is a genetic disease involving the loss of motor neurons in the spinal cord.1 Its …
ProteoNic Biosciences launches platform to enhance cell line productivity
September 17, 2025Research and Development Devices, ProteoNic Biosciences, cell line productivity, gene therapy
ProteoNic Biosciences, a biotech company headquartered in Leiden, Germany has unveiled its latest technology platform, the 2G UNic transposon system, developed …
Sartorius Stedim Biotech partners with Nanotein Technologies to develop cell therapy manufacturing
August 12, 2025Mergers and Acquisitions Corporate, Nanotein Technologies, Sartorius Stedim Biotech, cell therapy, gene therapy
Sartorius Stedim Biotech has partnered with Nanotein Technologies to expand access to immune cell activation reagents for cell therapy manufacturing. …
Amarna Therapeutics and NorthX Biologics collaborate to advance gene therapy for type 1 diabetes
July 10, 2025Mergers and Acquisitions, Research and Development Amarna Therapeutics, Diabetes, Immunology, NorthX Biologics, Type 1 diabetes, gene therapy, immune-mediated diseases
Amarna Therapeutics and NorthX Biologics have finalised an agreement to accelerate the development of Nimvec AM510, a gene therapy targeting …
Terumo BCT showcases new CAR-T workflow enabling faster cell expansion
June 20, 2025Manufacturing and Production CAR-T cell manufacturing, Devices, EULV, Eureka Biotechnology, Terumo Blood and Cell Technologies, cell therapy, eureka, gene therapy
Terumo Blood and Cell Technologies (Terumo BCT) has unveiled a new automated workflow that consolidates three critical CAR-T manufacturing steps …
First UK patient treated with CSL Behring’s gene therapy for Haemophilia B
June 20, 2025Research and Development CSL Behring, Haematology, NHS England, cell therapy, gene therapy, haemophilia B, prophylactic infusions
CSL Behring has announced that the first patient in the UK has received HEMGENIX (etranacogene dezaparvovec) through the NHS, marking …
Pfizer shares update on DMD gene therapy clinical trial
June 13, 2024Research and Development DMD, Musculo-skeletal disorder, Pfizer, duchenne muscular dystropy, gene therapy
Pfizer has announced an update from its phase 3 randomised, multicentre, double-blind, placebo-controlled CIFFREO trial, which assessed the investigational mini-dystrophin …
Vivet Therapeutics gains €4.9m for gene therapy development
February 2, 2024Research and Development CTX, Neurology, Vivet Therapeutics, funding, gene therapy
Vivet Therapeutics has announced that it has received €4.9m financing from the French Government for the advanced development of a …
Evox Therapeutics enters agreement for next generation gene therapy for heart disease
August 4, 2023Research and Development Cardiology, Evox Therapeutics, Icahn Mount Sinai, cvd, gene therapy
Evox Therapeutics has announced a research collaboration and option agreement with the Icahn School of Medicine at Mount Sinai (Icahn …
SNIPR Biome reports positive findings for first-in-human, CRISPR-based microbial gene therapy
May 31, 2023Research and Development Infections and infestations, SNIPR Biome, clinical trial, crispr, gene therapy
Danish CRISPR-based microbial gene therapy company SNIPR Biome have announced positive interim data from its phase 1 clinical trial with …
Rocket Pharmaceuticals granted FDA RMAT designation for gene therapy
May 24, 2023Medical Communications FDA, Haematology, RMAT, Rocket Pharmaceuticals, gene therapy
Rocket Pharmaceuticals, a US-based biotechnology company, has announced that it has received US Food and Drug Administration (FDA) Regenerative Medicine …
Krystal Biotech secures FDA approval for redosable topical gene therapy
May 22, 2023Medical Communications FDA, Krystal Biotech, Paediatrics, dystrophic epidermolysis bullosa, gene therapy
US-based biotech company Krystal Biotech has announced that the US Food and Drug Administration (FDA) has approved its topical gene …
Nemours Children’s Health selected to conduct first gene therapy for Morquio A syndrome
May 17, 2023Research and Development Morquio A syndrome, NIH, Nemours Children's Health, Paediatrics, gene therapy
Nemours Children’s Health, based in Delaware, US, has been chosen by the Foundation for the National Institutes of Health (FNIH) …
FDA pre-meeting report raises concerns over Sarepta’s gene therapy for Duchenne muscular dystrophy
May 11, 2023Medical Communications Duchenne Muscular Dystrophy, FDA, Musculo-skeletal disorder, Sarepta Therapeutics, gene therapy
The US Food and Drug Administration (FDA) has released a report in preparation for its 12 May decision on Sarepta …
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