Tag: Gene Therapy

Pfizer Announced Updated Results from Study Evaluating SB-525 Gene Therapy for Hemophilia A

Sangamo Therapeutics, Inc., a genomic medicine company, and Pfizer, Inc. announced updated results from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A....

Strategic Partnership for Gene Therapy Development Announced by Amicus and Catalent

gene-therapy
Amicus Therapeutics, Inc. has entered into a strategic manufacturing agreement with Paragon Gene Therapy, a unit of Catalent Biologics, for clinical manufacturing capabilities and capacity for multipl...

Strategic Gene Therapy Licensing Agreement Announced by PTC and Odylia

PTC Therapeutics, Inc. and Odylia Therapeutics announced a strategic collaboration to develop novel gene therapies in rare inherited retinal diseases (IRDs) utilizing the Anc80 vector system developed...

Oxford Biomedica and Santen Plan to Develop Gene Therapy Vectors for Inherited Retinal Disease

gene therapy
Oxford Biomedica plc, a leading gene and cell therapy group, and Santen Pharmaceutical Co., Ltd., the market leader for prescription ophthalmic pharmaceuticals in Japan with a global presence in over ...

Axovant Gene Therapies Announce Strategic Partnership with Yposkesi

gene therapy
Axovant Gene Therapies Ltd., a clinical-stage company developing innovative gene therapies, announces it has signed a strategic partnership with Yposkesi, a leading Contract Development and Manufactur...

Biogen Completed Acquisition of Nightstar Therapeutics Worth $800m

biogen
Biogen announced that it has completed its acquisition of Nightstar Therapeutics, a clinical-stage gene therapy company, which is focused on adeno-associated virus (AAV) treatments for inherited retin...

bluebird bio Drug for Severe Genetic Disease Approved by EC

bluebird bio, Inc. announced that the European Commission (EC) has granted conditional marketing authorization for ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene), a gene therapy for p...

Sensorion Signed Agreement with Institut Pasteur on Gene Therapy Programs

gene therapy
Sensorion, a pioneering clinical-stage biopharmaceutical company which specialises in the development of novel therapies to restore, treat and prevent inner ear diseases such as hearing loss, tinnitus...

FDA Approved First Gene Therapy to Treat Children with SMA

The U.S. Food and Drug Administration approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA...

ElevateBio Gets $150M to Launch Cell and Gene Therapy Incubator

gene-therapy
ElevateBio, a Cambridge-based biotechnology holding company, today announced operations to create and build a broad portfolio of cell and gene therapy companies through partnerships with leading acade...

Neurogene to Advance Multiple Gene Therapy Programs

Neurogene, Inc., a company founded with a mission to bring life-changing medicines to patients and families affected by rare neurological diseases, announced a $68.5 million Series A financing. Invest...

B-MoGen and CytoSen to Develop NK Therapies

B-MoGen Biotechnologies, Inc. and CytoSen Therapeutics announced today a research collaboration to develop the next generation of gene-modified Natural Killer Cell (NK) therapies. B-MoGen will utilize...

CRISPR and ProBioGen to Develop Novel Technologies

CRISPR Therapeutics, a biopharmaceutical company focused on creating gene-based medicines for serious diseases, and ProBioGen, a German service and technology provider, have entered a multi-year resea...

Big Pharma’s Experts Go to Biotech

Three more big pharma R&D names have made the jump to biotech as Voyager Therapeutics, ImmunSYS and Immunocore announce they have appointed former execs from Roche, Novartis and AstraZeneca. Vo...

UK Gene Therapy Show Positive Results for Rare Skin Disease

gene-therapy
A gene therapy applied to the skin, developed by UK company Amryt Pharma, showed positive preclinical results for the treatment of a rare genetic skin disease. The disease, called recessive dystrop...

British Companies to Develop RALA Gene Therapy Delivery Method

cells
British companies promote the RALA gene therapy delivery method to the immunotherapy market. A collaboration between two companies has been formed in the UK to increase access and decrease the cost...