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.
Waters Corporation has officially completed its acquisition of Wyatt Technology, a leading provider of light scattering and field-flow fractionation instruments, software, accessories, and services.
Hansa Biopharma and Genethon have entered a research and development collaboration to test imlifidase as a gene therapy treatment in patients with pre-existing neutralizing antibodies.
BioMarin Pharmaceutical has opened the expansion of its manufacturing plant at Shanbally, Co. Cork: with the site now able to provide end-to-end manufacturing for a number of the company’s commercial products.
bluebird bio has announced the submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD).
Ginkgo acquires StrideBio's adeno-associated virus (AAV) capsid discovery and engineering platform assets and as well as gaining ownership of a preclinical asset.
In an effort to improve its cash runway, the UK biotech company Freeline Therapeutics has paused the development of a gene therapy candidate for the rare metabolic condition Fabry disease. Instead, the company will refocus its efforts on a gene therapy...
Remedium Bio, a US biotech, and Exothera, a Belgian CDMO, have established a collaborative agreement in relation to the US firm’s disease modifying gene therapy for osteoarthritis, AAV2-FGF18.
Novartis has presented new data which ‘underscore the transformational and sustained benefit’ of spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec): with treated children maintaining motor milestones after 7.5 years.
Intergalactic Therapeutics, a startup focused on non-viral gene therapy, reports positive preclinical results for its lead program, IG-002, addressing all forms of ABCA4-related retinopathies.
Evonik has opened a new GMP facility to manufacture lipids for advanced pharmaceutical drug delivery applications, which is situated at the company’s site in Hanau, Germany.
Spark Therapeutics, a Roche company, has officially started work on its US$575m gene therapy innovation center in West Philadelphia. The center is expected to be completed by 2026.
Three months after receiving the US green light, CSL’s candidate Hemgenix has become the first gene therapy to gain EU approval for the treatment of hemophilia B.
In 2022, the European Medicines Agency (EMA) recommended 89 medicines for marketing authorization: including six advanced therapy medicinal products and eight biosimilars.
The Cell and Gene Therapy Catapult (CGT Catapult) has confirmed its involvement in the development of a new £900m ($1,097m) life science campus in Stevenage, UK: which is set to become one of the largest in Europe.
