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Category: Cell and Gene Therapy


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

Cellular Origins, ScaleReady Partner to Improve Cell Therapy Manufacturing

May 11, 2023

Via: Contract Pharma

Cellular Origins, a TTP Company focused on enabling scalable, cost-effective, and efficient manufacture of cell and gene therapies (CGTs), entered into a collaborative partnership with ScaleReady, a joint venture between Bio-Techne, Fresenius Kabi and Wilson Wolf that brings together process […]


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 […]


Cell and Gene Therapy, Industry

Convergent raises $90M to develop its radiopharmaceutical for prostate cancer

May 5, 2023

Via: Biopharma Dive

While radiopharmaceuticals can be difficult to manufacture, they are an attractive way to more selectively direct radiation to tumor cells while sparing healthy tissue. Prostate cancer in particular has been a target of choice for radiopharmaceutical developers looking to add […]


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 […]


Cell and Gene Therapy, Industry

Challenges of Expanding CAR-T Cell Therapy into Solid Tumors

May 3, 2023

Via: Biopharm International

Since the first FDA approval in 2017, chimeric antigen receptor T-cell (CAR-T) therapy has garnered excitement as a potentially curative option for hard-to-treat blood cancers (1). In this therapy, T cells are genetically engineered to recognize specific tumor-surface antigens. When […]


Cell and Gene Therapy, Industry

J&J, building on CAR-T success, strikes another cell therapy deal

May 2, 2023

Via: Biopharma Dive

Once a latecomer to cell therapy research, J&J has established itself as a major player in recent years. A 2017 licensing deal with Chinese drug developer Legend Biotech yielded Carvykti, which has since become one of only two cell therapies […]


Cell and Gene Therapy, Industry

FDA Approves Drug for Rare Form of ALS

April 26, 2023

Via: Drugs.com

The U.S. Food and Drug Administration on Tuesday approved a new drug for people who have a rare, inherited type of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The medication, toferson (Qalsody), targets a mutation in the […]


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

FDA Approves Cord Blood Stem Cell Product, Omisirge, for Blood Cancer Patients

April 24, 2023

Via: Drugs.com

The U.S. Food and Drug Administration approved Omisirge (omidubicel-onlv), a substantially modified allogeneic cord blood-based cell therapy, to cut the risk of infection in patients with blood cancer following a myeloablative treatment, such as radiation or chemotherapy. Omisirge is administered […]


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

FDA approves Gamida’s cell therapy for blood cancer patients

April 19, 2023

Via: PMLiVE

Omisirge is specifically indicated for individuals aged 12 years and older who are planned for umbilical cord blood transplantation following a myeloablative conditioning regimen, such as radiation or chemotherapy. Stem cell transplantation is a common treatment for blood cancers, which […]


Cell and Gene Therapy, Industry

J&J study data show dramatic benefit to multiple myeloma cell therapy

April 19, 2023

Via: Biopharma Dive

The cancer cell therapy Carvykti dramatically outperformed standard drugs in a late-stage clinical trial testing its use in earlier treatment of the blood cancer multiple myeloma, according to data from a study abstract that was briefly posted online Tuesday. The […]


Cell and Gene Therapy, FDA, Industry, Regulations

FDA approves cell therapy for patients with blood cancers to reduce risk of infection following stem cell transplantation

April 17, 2023

Via: World Pharma News

Today, the U.S. Food and Drug Administration approved Omisirge (omidubicel-onlv), a substantially modified allogeneic (donor) cord blood-based cell therapy to quicken the recovery of neutrophils (a subset of white blood cells) in the body and reduce the risk of infection. […]