Category: Cell and Gene Therapy

September 26, 2022

The phase 3 AFFINE trial was paused voluntarily by the two partners almost a year ago, and subsequently placed on hold by the FDA, after patients treated with the therapy produced higher than expected levels of Factor VIII – the […]

September 23, 2022

Pfizer and Sangamo Therapeutics will soon resume a Phase 3 trial of their gene therapy for the blood disorder hemophilia A, nearly a year after safety concerns forced its suspension. Specifically, the companies said Thursday evening they plan to resume […]

September 22, 2022

GenScript and Avectas agreed to partner to develop an improved non-viral cell therapy manufacturing process. By combining Avectas’ cell engineering technology with GenScript’s expertise in synthetic long oligo production, the partnership aims to demonstrate a novel and efficient solution for […]

September 20, 2022

GenScript – the life-sciences research tools and services provider – and Avectas, a cell engineering technology company, are partnering to develop an improved non-viral cell therapy manufacturing process. The two companies are focusing on new methods for developing cell therapies […]

September 19, 2022

Until this August, just two gene therapies for inherited diseases were available in the U.S. Now, in the span of one month, that count has doubled, with the Food and Drug Administration approving new treatments for a rare blood condition […]

September 14, 2022

The French biotech said the cash injection – which follows a €45 million Series A in 2020 – will also help it bring forward the first development candidate in a collaboration signed with gene-editing specialist Intellia Therapeutics last year, currently […]

September 13, 2022

Ori Biotech, a leader in cell and gene therapy (CGT) manufacturing technologies, and the Cell Therapy Manufacturing Center (CTMC), a joint venture between National Resilience Inc. and MD Anderson Cancer Center, have entered a collaborative partnership to develop industrial manufacturing […]

September 9, 2022

Researchers at the National Eye Institute (NEI), part of the National Institutes of Health, have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness […]

September 5, 2022

Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially protect diseased motor neurons in the spinal cord of […]

September 1, 2022

Sangamo Therapeutics is continuing to enroll and treat patients with a rare genetic condition called Fabry disease in an early clinical trial of a gene therapy it is developing, announcing Tuesday updated results from the first 11 study participants. Data […]

August 31, 2022

FDA officials are proceeding with caution as the gene editing field advances. In another recent example, the agency had issued a partial clinical hold to Editas Medicine before this July allowing that company to proceed with testing of a sickle […]

August 31, 2022

Cytiva and Forecyte Bio announced on August 25, 2022 that they will collaborate to accelerate the manufacturing and development of the cell and gene therapy industry in China and the United States. The companies will utilize Cytiva’s Fast Trak center […]

August 31, 2022

Mayo Clinic, Hibiscus BioVentures and Innoforce Pharma have launched Mayflower BioVentures, a cell and gene therapy accelerator dedicated to identifying and forming companies around technologies that address unmet patient needs. This academic and industry relationship will establish independent cell and […]

August 31, 2022

Applied StemCell, Inc. (ASC), a cell and gene therapy CRO/CDMO focused on supporting the research community and biotechnology industry for their needs in developing and manufacturing cell and gene products, is expanding its current good manufacturing (cGMP) facility. ASC has […]

August 26, 2022

The US biotech claimed conditional marketing approval from the European Commission for Roctavian (valoctocogene roxaparvovec) as a treatment for adults with severe haemophilia A, who don’t have a history of developing the antibodies that inhibit the activity of blood-clotting protein […]

August 18, 2022

Bluebird bio’s new gene therapy, approved in the U.S. Wednesday, offers patients with an inherited blood disorder a one-time, potentially curative, treatment option. But it will come at a price of $2.8 million, making the therapy the most expensive drug […]

August 18, 2022

Cellipont Bioservices, a cell therapy contract development and manufacturing organization (CDMO), has unveiled plans for a 76,000 square foot commercial-ready manufacturing facility for cell therapies and gene-modified cell therapies. Located in The Woodlands, TX, the facility will include state-of-the-art manufacturing, […]

August 16, 2022

Twelve years ago, Bill Ludwig and Doug Olson were running out of options when they enrolled in a clinical trial at the University of Pennsylvania. They had chronic lymphocytic leukemia, a type of blood cancer, and their disease had progressed […]

August 10, 2022

Biotechnology startup GentiBio on Wednesday struck a deal with Bristol Myers Squibb to develop a cell therapy for inflammatory bowel disease — the latest sign of the pharmaceutical industry’s interest in an emerging field of drug research. Through the alliance, […]

August 1, 2022

The US biotech – which already has three antisense drugs for DMD on the market – had said earlier it was planning to delay the application into 2023, so the new announcement marks an acceleration of its plans. Sarepta reported […]