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Category: Cell and 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 […]


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 […]


Cell and Gene Therapy, Industry

Working Back Towards Robust Cell Therapy Manufacturing

December 28, 2021

Via: Genetic Engineering and Biotechnology News

The general notion regarding autologous manufacturing is that it has huge challenges, points out Parvathi Chandran, senior manager for research strategy and communication at Regrow Biosciences. “But our experience is that you can work backwards, adapting your manufacturing process, and […]


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 […]


Cell and Gene Therapy, Industry

J&J cell therapy head exits to take up post at fledgling biotech

December 1, 2021

Via: FierceBiotech

Johnson & Johnson has lost its global head, cell therapy platform and discovery. Tamas Oravecz, Ph.D., took up the post at J&J’s Janssen earlier this year, only to leave to become chief scientific officer at the newly minted cancer biotech […]


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 […]


Cell and Gene Therapy, Industry

Cell and gene therapy player CBM plots expansion, 2,000 new hires with funding from South Korean partner

November 16, 2021

Via: FiercePharma

The Center for Breakthrough Medicines (CBM), a CDMO founded in 2019, is getting financial backing from South Korean financial group SK Inc. to expand its manufacturing capacity. The two companies didn’t disclose a dollar amount, but CBM said it will […]


Cell and Gene Therapy, Industry

Protagonist posts phase 2 hormone replacement data, pitching for lead role in genetic iron overload disease

November 15, 2021

Via: FierceBiotech

Protagonist Therapeutics wants to take the lead in a genetic iron disease, delivering midphase data on a hormone replacement therapy it envisages offering an alternative to blood draws. Rusfertide is Protagonist’s attempt to improve on the druglike properties of the […]