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gene therapy

Showing 15 posts of 52 posts found.

Rare diseases – spinal muscular atrophy

January 30, 2026
Medical 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 …

Feature

Five Facts about spinal muscular atrophy

January 28, 2026
Medical 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 …

Feature

ProteoNic Biosciences launches platform to enhance cell line productivity

September 17, 2025
Research 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 …

News
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Sartorius Stedim Biotech partners with Nanotein Technologies to develop cell therapy manufacturing

August 12, 2025
Mergers 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. …

News

Amarna Therapeutics and NorthX Biologics collaborate to advance gene therapy for type 1 diabetes

July 10, 2025
Mergers 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 …

News
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Terumo BCT showcases new CAR-T workflow enabling faster cell expansion

June 20, 2025
Manufacturing 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 …

News

First UK patient treated with CSL Behring’s gene therapy for Haemophilia B

June 20, 2025
Research 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 …

News

Pfizer shares update on DMD gene therapy clinical trial

June 13, 2024
Research 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 …

News
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Vivet Therapeutics gains €4.9m for gene therapy development

February 2, 2024
Research 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 …

News

Evox Therapeutics enters agreement for next generation gene therapy for heart disease

August 4, 2023
Research 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 …

News

SNIPR Biome reports positive findings for first-in-human, CRISPR-based microbial gene therapy

May 31, 2023
Research 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 …

News

Rocket Pharmaceuticals granted FDA RMAT designation for gene therapy

May 24, 2023
Medical 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 …

News
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Krystal Biotech secures FDA approval for redosable topical gene therapy

May 22, 2023
Medical 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 …

News

Nemours Children’s Health selected to conduct first gene therapy for Morquio A syndrome

May 17, 2023
Research 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) …

News

FDA pre-meeting report raises concerns over Sarepta’s gene therapy for Duchenne muscular dystrophy

May 11, 2023
Medical 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 …

News
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