Manufacturing, Gene Therapies

The companies will offer an IT platform that enhances the visibility and performance of the overall personalized medicine treatment process.

Researchers at the University of Delaware have made a step forward in gene therapy by engineering microparticles that deliver gene-regulating material to hematopoietic stem and progenitor cells, which live deep in bone marrow and direct the formation of blood cells.

The partnership provides Sarepta with capacity and manufacturing slots for GMP-grade plasmid production for its micro-dystrophin Duchenne muscular dystrophy gene therapy program, as well as plasmid capacity for future gene therapy programs.

A new facility in California will expand Orchard Therapeutic’s capacity to develop and deliver lentiviral vector and gene-corrected hematopoetic stem cells.

Cobra, Pall, and Cell and Gene Therapy Catapult are collaborating to develop continuous manufacturing for gene therapy production.

Moving forward with gene therapy development requires a “quantum leap” in manufacturing capabilities.

Improving the manufacturing of gene therapy vectors will be crucial to making advanced treatments accessible to more patients who need them, agreed panelists at the 2018 Galien Forum.

The company will collaborate with GlycoBac to offer an insect cell line for the development of viral vaccines and gene therapies.

The European Medicines Agency has recommended Luxturna (voretigene neparvovec) as the first treatment option for hereditary retinal dystrophy with mutations of the RPE65 gene.

More consistent and reliable production processes are critical for advancing innovative treatments.

The acquisition will strengthen Astellas Pharma’s position in ophthalmology.

Boehringer Ingelheim joins Oxford BioMedica, UK Cystic Fibrosis Gene Therapy Consortium, and Imperial Innovations to form a partnership for developing a new gene therapy to treat cystic fibrosis.

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.