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Tag: gene therapy


FDA, Regulations

FDA delays decision on Duchenne gene therapy and considers narrower approval

May 24, 2023

Via: Biopharma Dive

The Food and Drug Administration has delayed its review of a closely watched gene therapy for Duchenne muscular dystrophy by one month, taking more time to consider whether approval should be initially limited to children who are most likely to […]


Cell and Gene Therapy, Industry

FDA approves Krystal gene therapy for rare wound disorder

May 22, 2023

Via: Biopharma Dive

Dystrophic epidermolysis bullosa is one of four main types of the disorder. Depending on the inheritance pattern, the condition can be severe and disabling, causing blisters anywhere on the skin from minor trauma and friction. The form Vyjuvek is cleared […]


Cell and Gene Therapy, Industry

Siren emerges from stealth with plans to marry gene therapy to cancer immunotherapy

May 17, 2023

Via: Biopharma Dive

Two and a half years ago, Nicole Paulk’s coworkers told her to start a company. The gene therapy researcher and then University of California, San Francisco assistant professor had just showed them data from mice studies testing a new kind […]


Cell and Gene Therapy, Industry

CATTI Opens Cell and Gene Therapy Training Center of Excellence

May 16, 2023

Via: Contract Pharma

The Canadian Advanced Therapies Training Institute (CATTI) Inc. launched its first in-person training site at the University of Guelph through a partnership with OmniaBio Inc., CCRM and the university. The training program, to begin in summer 2023, will cater to […]


Cell and Gene Therapy, Industry

Biotech startup Ray raises $100M for vision-restoring gene therapies

May 16, 2023

Via: Biopharma Dive

Diseases of the eye have been a popular target for gene therapy development, due in part to the potential for efficient delivery via injection as well as the lower risk of dangerous immune responses. Luxturna, the first gene therapy for […]


Cell and Gene Therapy, Industry

Life Biosciences, Forge Biologics Partner for Manufacturing to Advance Gene Therapies

May 15, 2023

Via: Biopharm International

Life Biosciences, a biotechnology company, and Forge Biologics, a genetic medicines manufacturing organization, announced on May 8, 2023 that they are in a manufacturing partnership to help advance Life Bio’s partial epigenetic reprogramming platform to further address aging-related diseases, according […]


Cell and Gene Therapy, Industry

FDA panel narrowly backs approval of Sarepta’s Duchenne gene therapy

May 12, 2023

Via: Biopharma Dive

A group of Food and Drug Administration advisers narrowly supported approving what could be the first gene therapy for Duchenne muscular dystrophy in a meeting Friday, clearing the way for the agency to make a closely watched decision later this […]


Cell and Gene Therapy, Industry, News

FDA staff skeptical of Sarepta’s Duchenne gene therapy, documents show

May 10, 2023

Via: Biopharma Dive

Food and Drug Administration scientists evaluating what could be the first gene therapy for Duchenne muscular dystrophy appear skeptical of the treatment’s benefit, adding to questions about its approval prospects days before a crucial regulatory meeting. On Friday, the FDA […]


Cell and Gene Therapy, Industry

OBiO, Chinagene Sign Gene Therapy Manufacturing Services Agreement

May 8, 2023

Via: Contract Pharma

OBiO Technology Corp., a provider of gene and cell therapy-focused biotechnology, entered into a strategic commercial manufacturing collaboration with Chinagene, a developer of clinical genetic diagnosis, and gene therapy drugs. Under the agreement, OBiO will provide contract development and manufacturing […]


FDA, Regulations

FDA Approves First Monoclonal Antibody Treatment, Librela, for Arthritis in Dogs

May 8, 2023

Via: Drugs.com

Man’s aging best friend has a new treatment to dull osteoarthritis pain as the U.S. Food and Drug Administration (FDA) announced approval Friday of the first monoclonal antibody for dogs. Called Librela, the bedinvetmab shot controls pain from the most […]


Cell and Gene Therapy, Industry

Forge Biologics Partners with Labcorp on AAV Gene Therapy Development

May 3, 2023

Via: Contract Pharma

Forge Biologics, a manufacturer of genetic medicines, will collaborate with Labcorp, a global life sciences company, on gene therapy development and manufacturing. Under the alliance, gene therapy clients will have access to manufacturing capabilities, drug development services, and coordinated scientific […]


Clinical Trials, Research and Development

Flatiron, Sanofi Partner on Efficiency in Clinical Trials

May 1, 2023

Via: Contract Pharma

Flatiron Health is collaborating with Sanofi to redesign the clinical trial experience for sponsors, sites, and patients in the therapeutic area of oncology. The multi-study collaboration will focus on improving clinical trial data acquisition, delivery, and quality through site-facing technology […]


Cell and Gene Therapy, Industry

bluebird bio submits US application for sickle cell gene therapy

April 26, 2023

Via: PMLiVE

The application, which includes a request for priority review, is specifically for lovotibeglogene autotemcel (lovo-cel) to treat SCD patients aged 12 years and older who have a history of painful complications associated with the disease. Affecting approximately 100,000 people in […]


Cell and Gene Therapy, Industry

French biotech pulls EMA application for eye disease gene therapy

April 21, 2023

Via: Biopharma Dive

GenSight is facing another setback and a dwindling cash pile at an unfortunate time. A market downturn has made it difficult for drug developers to raise money by selling shares. For GenSight to continue its clinical work, it will likely […]


Cell and Gene Therapy, Industry

A new biotech wants to ease a bottleneck in cell and gene therapy production

April 11, 2023

Via: Biopharma Dive

A biotechnology company looking to alleviate a problem in cell and gene therapy production emerged from “stealth” mode on Tuesday, unveiling itself alongside $64 million in funding. Based in Philadelphia, VintaBio was founded by Junwei Sun and Shangzhen Zhou, two […]


Cell and Gene Therapy, Industry

New therapy harnesses patients’ blood cells to fight tumors

April 10, 2023

Via: World Pharma News

Adoptive cell therapy (ACT) has become a promising immunotherapy tool to help treat advanced melanoma. The therapy, which harnesses immune cells collected from the patient’s own tumors, could provide a new treatment option to cancer patients, potentially bypassing radiation therapies […]


Cell and Gene Therapy, Industry

Freeline cuts more jobs and halts Fabry gene therapy work

April 4, 2023

Via: Biopharma Dive

Freeline’s retrenchment adds to the growing list of reorganizations, job cuts and other strategic maneuvers young drugmakers have pursued amid a sharp downturn in the public markets. Already this year, at least 56 biotech companies have announced layoffs, according to […]


Cell and Gene Therapy, Clinical Trials, Industry, Research and Development

Changing cell & gene therapy landscape and implications on clinical trials

April 3, 2023

Via: Biopharma Dive

Cell and gene therapy clinical research is a field that holds promise for the treatment of many diseases. These therapies involve using living cells or genes to treat or prevent diseases and have the potential to revolutionize how we approach […]


FDA, Regulations

Bluebird, awaiting FDA feedback, says it’s likely to miss filing goal for sickle cell gene therapy

March 29, 2023

Via: Biopharma Dive

Bluebird is now several months into launching two rare disease gene therapies for which it won FDA approval last fall. But its progress on that front was overshadowed Wednesday by news of the delay for the company’s sickle cell treatment, […]


Cell and Gene Therapy, Industry

Addressing manufacturing challenges in gene therapy development

March 20, 2023

Via: Biopharma Dive

Gene therapy is on the rise. As clinical success, innovation and investment in this area strengthens, Precedence Research predicts that the cell and gene therapy market will grow at a CAGR of 22 percent by 2030. Additionally, the FDA expects […]