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Tag: gene therapy


Cell and Gene Therapy, Industry

New Gene Therapy May Potentially Treat Severe Form of Epilepsy

January 20, 2022

Via: Genetic Engineering and Biotechnology News

Scientists from the University of Virginia (UVA) School of Medicine describe in a study involving mouse models how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the […]


Cell and Gene Therapy, Industry

CBM and Penn Strike $100M Cell & Gene Therapy Deal

January 20, 2022

Via: Contract Pharma

The Center for Breakthrough Medicines (CBM) and the University of Pennsylvania’s Gene Therapy Program (GTP) have signed a landmark collaboration agreement, to combine Penn’s knowledge and expertise in the development of Gene therapies with CBM’s manufacturing capacity and singular focus […]


Manufacturing, Research and Development

Ori Biotech reaps $100M in series B round to help launch innovative cell manufacturing tech

January 18, 2022

Via: FiercePharma

Ori Biotech, which is on the cusp of launching an innovative process for manufacturing cell and gene therapies (CGTs), tallied more than $100 million in its latest funding round. The company will use the money to add key personnel in […]


Cell and Gene Therapy, Industry

Pfizer expands into gene editing with Beam research deal

January 10, 2022

Via: Biopharma Dive

Last summer, Mikael Dolsten, head of research and development at Pfizer and the company’s top scientist, was thinking of new uses for the messenger RNA technology the pharmaceutical giant had developed so effectively for its coronavirus vaccine. The drugmaker already […]


Cell and Gene Therapy, Industry

Biogen brings in a successor to Spinraza

January 4, 2022

Via: Biopharma Dive

First approved in late 2016, Spinraza has become one of the most important medicines in Biogen’s arsenal, accounting for about a fifth of the company’s total product revenue. Recently, though, the Spinraza franchise hasn’t fared as well due to competitive […]


Cell and Gene Therapy, Industry

Avrobio stops work on rare disease gene therapy after unexpected study results

January 4, 2022

Via: Biopharma Dive

Fabry is one of several dozen diseases known collectively as lysosomal storage disorders, all caused by genetic breakdowns in how the body’s cells process complex fats or sugars. Treatment, when it’s available, usually involves infusions of the digestive proteins that […]


FDA

FDA sets April date for verdict on Incyte’s vitiligo drug

December 15, 2021

Via: Pharmaphorum

The FDA has kicked off a priority review of Opzelura (ruxolitinib), a JAK1/JAK2 inhibitor it approved to treat atopic dermatitis in September, and aims to decide on the vitiligo indication by 18 April next year. If approved, Opzelura would provide […]


Clinical Trials, Research and Development

Sotio Biotech, MSD Enter Clinical Trial Collaboration & Supply Agreement

December 8, 2021

Via: Contract Pharma

Sotio Biotech, a clinical stage immuno-oncology company owned by PPF Group, has entered into a clinical trial collaboration and Supply Agreement with MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ., through its subsidiaries, to evaluate the combination of […]


Cell and Gene Therapy, Industry

Astellas and Dyno Therapeutics to develop AAV gene therapy vectors

December 6, 2021

Via: European Pharmaceutical Review

Astellas Pharma and Dyno Therapeutics have announced an option and license agreement to develop next-generation adeno-associated virus (AAV) vectors for gene therapy directed to skeletal and cardiac muscle using Dyno’s CapsidMap™ platform. Dyno’s CapsidMap platform represents a new approach applying […]


Cell and Gene Therapy, Industry

Dyno attracts $1.6bn gene therapy deal with Astellas

December 2, 2021

Via: Pharmaphorum

The Japanese drugmaker is paying $18 million upfront to exercise an option on the AAV platform – which my help avoid safety issues that have emerged with other AAV-based gene therapies – with milestone and royalty payments that could exceed […]


FDA, Industry, Regulations, Vaccines

Pfizer, Moderna score FDA nods for COVID boosters in all adults, setting up high-stakes CDC meeting

November 19, 2021

Via: FiercePharma

The race between Moderna and Pfizer to gain FDA nods for their COVID-19 vaccines as boosters in all adults has ended in a dead heat. On Friday morning, the FDA simultaneously expanded emergency use authorizations for both mRNA vaccines to […]


Cell and Gene Therapy, Industry

Gene Therapy Could Be Big Advance Against Hemophilia

November 18, 2021

Via: Drugs.com

Gene therapy shows promise in reducing, and even halting, potentially life-threatening bleeding events in people with hemophilia, researchers report. Hemophilia A is the most common inherited bleeding disorder, affecting one in 5,000 males worldwide. It’s caused by a missing coagulation […]