Tag: Gene Therapy

Audentes Plans to Invest over $100 Million and Create over 200 New Jobs in Lee County

Audentes Therapeutics, an Astellas company focused on developing innovative AAV-based genetic medicines, announced that it plans to invest $109 million to build a new 135,000 square foot, state-of-the...

FDA Continues Support of Innovation in Development of Gene Therapy Products

This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA h...

Krystal Biotech Breaks Ground On Second Gene Therapy Manufacturing Facility

Krystal Biotech, Inc., a gene therapy company developing medicines to treat rare diseases, announced the ground breaking of the second commercial gene therapy facility in Findlay Township, Pennsylvani...

UK Accounts for Over 12% of Global Cell and Gene Therapy Clinical Trials

The Cell and Gene Therapy Catapult (CGT Catapult) released their 2019 UK advanced therapies medicinal products (ATMPs) clinical trials database showing that the 127 ongoing trials represent a ~45% inc...

EMA Validated BioMarin’s Application to Market Investigational AAV Gene Therapy

gene
The European Medicines Agency (EMA) has validated BioMarin’s application to market its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A. As such, the compa...

FerGene Announced Positive Results from Phase 3 Clinical Trial of Nadofaragene Firadenovec

gene-editing
FerGene, a new gene therapy company formed by Ferring Pharmaceuticals and Blackstone Life Sciences, announced positive results from the pivotal Phase 3 clinical trial evaluating nadofaragene firadenov...

Pfizer, Novartis and 11 Drugmakers Set Aside $2bn to Invest in Gene Therapy Manufacturing

gene therapy
Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control the ...

Blackstone Group Ready to Invest $400m in JV with Ferring

Blackstone Group Inc said it will invest $400 million in a joint venture with Swiss drug company Ferring that is working on an experimental gene therapy for bladder cancer, the private equity giant’s ...

FUJIFILM Diosynth Announced Gene Therapy Services Expansion

gene
FUJIFILM Diosynth Biotechnologies (FDB), a leading global biologics Contract Development and Manufacturing Organization (CDMO) has announced the expansion of its gene therapy services with the additio...

The Russian Center for Precision Genome Editing Will Develop Drugs for Autoimmune Pathologies

The Russian Center for Precision Genome Editing and Genetic Technologies for Biomedicine, which has become one of the three centers for genomic research created within the framework of the national pr...

Growth of Gene Therapies for Neurology Indications Predicted by GlobalData

A new report has predicted that gene therapy development will pick up the pace but a high price point continues to pose a challenge. According to a new report, ongoing collaborations between ...

Wize Pharma Announced Exclusive License Agreement with Copernicus Therapeutics

Wize Pharma, a clinical-stage biopharmaceutical company focused on the treatment of ophthalmic disorders, announced it has entered into an Exclusive License Agreement with Copernicus Therapeutics, pur...

New Platform for Gene Therapies Developed at the University of Wisconsin-Madison

Alternative viral delivery of gene therapy developed. New nanocapsules are a promising platform for many types of gene therapies due to their small size and superior stability. Researchers at...

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...