The European Commission has granted Orphan Drug Designation for Vivet Therapeutics’ gene therapy product VTX-806 for the treatment of Cerebrotendinous Xanthomatosis (CTX).
Pfizer will let go of 150 employees at its Sanford, US site that were involved in the development of a gene therapy for Duchenne muscular dystrophy that recently failed in a phase 3 clinical trial.
A gene therapy being developed by Lexeo Therapeutics to treat cardiomyopathy caused by Friedreich ataxia, a rare neurodegenerative genetic disease, has achieved good interim results in a phase 1/2 trial.
Thermo Fisher’s cutting-edge ultra-cold facility in Bleiswijk, Netherlands, aims to accelerate advanced therapies development by offering comprehensive cold and ultra-cold services.
Andelyn Biosciences, a leading cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been chosen by UMass Chan Medical School to manufacture clinical-grade AAV9-CSA vector using its suspension AAV Curator Platform...
RNA therapeutics have emerged as a transformative approach to treating many conditions, but the industry still typically relies on inefficient batch manufacturing methods.
Hot on the heels of last year’s EU approval of Hemgenix, the first gene therapy for the blood disorder hemophilia B, the UK and Denmark are the latest European nations to take steps to pay for the treatment.
The US FDA has delayed approval of Rocket Pharmaceuticals’ gene therapy for treatment of a rare immune condition known as leukocyte adhesion deficiency, citing a need for more information about the manufacturing process.
Thermo Fisher’s new state-of-the-art ultra-cold facility in Bleiswijk, Netherlands, aims to accelerate advanced therapies development by offering a comprehensive range of cold and ultra-cold services.
Regeneron Pharmaceuticals’ gene therapy for otoferlin-related hearing loss, a genetic cause of deafness, has restored hearing to normal levels in one deaf child within just 24 weeks.
Circio, a biotech firm specializing in circular RNA-based gene therapy, has unveiled two posters showcasing in vivo proof-of-concept for its circVec platform at ASGCT 2024.
SK pharmteco has signed an agreement with Ferring Pharmaceuticals, to scale up commercial manufacturing capacity for the drug substance of Ferring’s intravesical gene therapy Adstiladrin.
Great Ormond Street Hospital (GOSH) has announced its new plan to ‘revolutionise’ how children living with rare diseases can gain access to cutting-edge gene therapy.
Dr. Claudia Zylberberg is a scientist and founder, board member and former CEO of Akron Biotech, a company that makes materials and technologies to support advanced therapy development. We spoke to her about her career, personal triumphs and candidacy...
The U.S. Food and Drug Administration has cleared AGC Biologics’ Milan site to begin manufacturing of Orchard Therapeutics’ Lenmeldy (atidarsagene autotemcel), a gene therapy for early-onset metachromatic leukodystrophy (MLD).
ReciBioPharm has partnered with pre-clinical gene therapy company GeneVentiv Therapeutics to advance development of an Adeno-Associated Virus (AAV)-based universal gene therapy for haemophilia, and the first to treat haemophilia patients with inhibitors.
Form Bio and Ginkgo Bioworkshave joined forces in what they call a groundbreaking partnership aimed at advancing AAV gene therapy design and development.
Neurogene, a clinical-stage company developing genetic medicines, is expanding its ongoing phase 1/2 clinical trial investigating NGN-401 as a treatment for female pediatric patients with Rett Syndrome.
OneChain Immunotherapeutics has received €1.9m from the Spanish Ministry of Science and Innovation to advance a ‘groundbreaking’ CAR-T therapy for acute lymphoblastic leukemia type T into clinical development.
A collaboration to expand autologous CAR-T programs into trials for multiple cancers in a cost-efficient way, has been formed by BioNTech SE and Autolus Therapeutics plc.
We sat down with Xiaojun Liu, director of AAV process development at ReciBioPharm, to discuss the growing need for carefully designed platforms to deliver safe and cost-effective AAV therapies to patients at pace.
Healthcare industry professionals scored cell and gene therapy (CGT) as the industry trend to have the greatest impact on the pharmaceutical industry in 2024, in a recent survey launched by data and analytics firm GlobalData.
Sarepta Therapeutics has hit a setback as its approved gene therapy Elevidys missed the primary goal of a phase 3 trial in children aged 4 through 7 years with the inherited disease Duchenne muscular dystrophy (DMD).
The London and New York-based gene therapy player MeiraGTx Holdings has received a $30 million investment from Sanofi in addition to potential strategic deals with the big pharma company down the road.
SpliceBio, a genetic medicines company harnessing protein splicing to develop the next generation of gene therapies, has partnered with Spark Therapeutics to develop a gene therapy for an inherited retinal disease.
Laverock Therapeutics, the gene editing-induced gene silencing platform for human therapeutic applications, has expanded its seed funding round to £13.5 million.
At Advanced Therapies Europe 2023, BioPharma Reporter caught up with ScaleReady's Jenny Stjernberg to discuss her work at the biotech company, her journey from academia to commercial and female representation in the cell and gene therapies industry....
TherageniX, a University of Nottingham spin out pioneering a dry powder gene therapy formulation for bone graft augmentation, has been awarded a £995,000 grant from Innovate UK.
Kadans Science Partner is set to welcome AviadoBio to its recently completed wet lab innovation and workspace centre at 20 Water Street in Canary Wharf.
Cell and Gene Therapy Catapult, Rentschler Biopharma, and Refeyn have announced a new partnership to develop and apply ‘innovative’ process analytical technologies (PAT) to improve the process and efficiency of AAV manufacture.
With so many advanced therapies on the horizon, the next few years are poised to be one of significant progress for patients with over 2000 cell and gene therapies in active clinical trials globally.
Teknova, a producer of life science reagents, has opened a new GMP-certified production facility which it claims will enable bioprocessing and gene therapy companies to get into the clinic faster.
Evox Therapeutics, an exosome therapeutics company, has announced a research collaboration and option agreement with the Icahn School of Medicine at Mount Sinai in New York, US.
A new genetic sequencing technology from Element Biosciences has helped researchers from the Translational Genomics Research Institute (TGen), part of the City of Hope, identify the likely genetic causes of disorders in six of nine children from Sonora,...
Dr. Theresa Heah is the CEO of Intergalactic Therapeutics, bringing over two decades of global leadership experience in ophthalmology drug development along with commercialization in early-stage, private-staged companies. We spoke to her about her background,...
Syncona has launched Beacon Therapeutics, a new ophthalmic gene therapy company with a purpose to restore and improve the vision of patients with retinal diseases, with £96 million Series A financing.
