Manufacturing, Gene Therapies

The agency is releasing six new draft guidances to provide a regulatory framework for handling gene therapies.

The gene therapy company is expected to invest $55 million in a new manufacturing facility that will produce therapies for rare neurological genetic diseases.

The new company will develop proprietary RNA-based therapeutics and will provide broad lentiviral development and manufacturing expertise and support.

Scientists at Washington University School of Medicine in St. Louis have developed a new method that could help increase the long-term effectiveness of gene therapy.

The new 300,000-square-foot facility is considered the largest dedicated cell and gene therapy manufacturing facility with fully integrated services.

The companies aim to develop a potential zinc finger protein transcription factor-based gene therapy for treating Lou Gehrig’s disease.

The companies will co-develop and co-commercialize the lead candidate generated from their earlier collaboration to treat genetic blood disorders.

Lonza has entered into strategic license agreements for exclusive rights to a gene-therapy platform for developing treatments for hearing and balance disorders.

The company has opened a new California facility that, combined with its existing site in Foster City, CA, will quadruple its laboratory footprint for gene therapy products.

The clinical-stage biopharmaceutical company has announced the opening of a new gene therapy manufacturing facility in Modiin, Israel, which is anticipated to be the production site of the company’s lead cancer drug candidate.

This approval marks the second gene therapy to be approved by FDA and the first to be approved for certain types of non-Hodgkin lymphoma.

The committee has voted unanimously to approve Spark Therapeutics’ gene therapy candidate, Luxturna (voretigene neparvovec), for treating a genetically inherited blindness.

The Cell and Gene Therapy Catapult, one of UK’s centers of excellence, has partnered with Japan’s Forum for Innovative Regenerative Medicine to boost their respective stances in the global cell and gene therapy landscape.

HTG, a company that provides molecular profiling services, expects to complete submission of its premarket approval application to FDA for an in vitro gene assay by 2018.

Pfizer will invest $100 million to expand its manufacturing facilities in Sanford, North Carolina.

BeiGene and the Guangzhou Development District have established a joint venture to build a biologics manufacturing facility in China.

There is much work to do to achieve efficient, cost-effective production processes.

Spark Therapeutics and Selecta entered a license agreement for Selecta’s gene-therapy co-administration technology.

Oxford Genetics has secured a GBP1 million capital from investment group Mercia Technologies to support its growth strategy in delivering new services for cancer-fighting gene-therapy technologies.

FDA’s new Office of Tissues and Advanced Therapies will oversee a growing range of cellular and genetic products.

Scientists from the Fred Hutchinson Cancer Research Center have developed a semi-automated benchtop system for the manufacture of gene therapy.

Oxford Genetics increased its laboratory space to 5700 sq. ft. with a move to a new location at Oxford Science Park.

The company established a dedicated research center in Bangalore, India for Amgen.

Takeda Pharmaceutical Company and enGene have entered into a strategic alliance to develop novel therapies for specialty gastrointestinal diseases.

Synpromics announced collaborations with Avalanche Biotechnologies and Applied Genetic Technologies Corporation to use synthetic promoters to develop gene therapies, including adeno-associated virus technology for treating eye diseases.

There’s renewed optimism in the biomedical research community that years of effort finally may begin to pay off for developing cutting-edge gene and cellular treatments for debilitating and life-threatening conditions. Jill Wechsler reports.

The exclusive manufacturing collaboration will establish production for adeno-associated virus gene-therapy treatments incorporating REGENXBIO’s NAV Technology.

Genzyme will partner with Voyager Therapeutics for the discovery, development, and commercialization of novel gene therapies for central nervous system disorders.

AstraZeneca will team up with various organizations to employ CRISPR technology for precise gene editing in recombinant cell lines.