Category: Cell and Gene Therapy

April 23, 2024

The Food and Drug Administration has rejected an experimental cell therapy for a rare skin condition, asking its developer, Abeona Therapeutics, to provide more information about how the treatment is made. Abeona on Monday said the FDA issued a so-called […]

April 22, 2024

CAR-T therapies are personalized treatments made from patients’ own immune cells. Their production is precisely choreographed, as frozen cells are brought to manufacturing hubs where they are engineered and expanded into a cancer-seeking drug product. It’s laborious, time-consuming and costly. […]

April 11, 2024

Drugmakers are moving quickly to study whether cell therapy, already a proven treatment for certain blood cancers, can tamp down autoimmune conditions, too. Lupus, a chronic disease that can cause inflammation and pain across the body, is one of the […]

April 2, 2024

Ginkgo Bioworks is expanding its ability to help other companies make cell and gene therapies, this time by acquiring a startup with tools to improve cell-based treatments. Ginkgo on Tuesday announced it has bought Modulus Therapeutics, a Seattle-based biotechnology startup. […]

March 26, 2024

The Dallas, Texas-based biotech said it plans to submit a dossier to the FDA later this year after its MCO-010 (sonpiretigene isteparvovec) gene therapy achieved almost all its primary and secondary efficacy objectives in the phase 2b RESTORE trial. Approximately […]

March 20, 2024

Gene therapy makers like Orchard are counting on insurers to recognize the value proposition of their products. Unlike traditional pharmaceuticals that might be taken regularly for the rest of a person’s life, gene therapies carry the promise of a long-lasting […]

March 6, 2024

Affecting an estimated one in 10,000 infants globally, SMA is a rare, genetic neuromuscular disease and a leading genetic cause of infant death. Caused by the lack of a functional SMN1 gene, the most severe forms of SMA result in […]

March 1, 2024

BioCina, a biologics contract development and manufacturing organization (CDMO) and NovaCina, a global fill-and-finish CDMO, have announced a partnership to offer integrated drug substance and drug product solutions for biologics developers. This will allow BioCina to provide fill/finish services while […]

February 29, 2024

Kenai Therapeutics, a San Diego-based biotechnology company, has raised $82 million to support its work developing cell therapies for nervous system disorders. Cure Ventures, a new venture capital firm founded by three longtime biotech investors, co-led the Series A round […]

February 27, 2024

Bayer announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for BAY 2927088 for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, […]

February 23, 2024

Roctavian’s approval was a scientific milestone, the culmination of years of research developing a genetic medicine for the chronic blood disease hemophilia A. Its launch, which began in Europe in 2022 and the U.S. last year, has been a focus […]

February 23, 2024

The regulator’s decision means that RRMM patients who have achieved a complete response or better for at least six months can receive a 1.5mg/kg dose of the BCMA-targeting bispecific antibody every two weeks. More than 35,000 new cases of multiple […]

February 21, 2024

A new research paper offers the most thorough look yet at a study that sparked the drug industry’s recent experimentation with cell therapy for autoimmune diseases. The paper, which was published in The New England Journal of Medicine on Wednesday, […]

February 20, 2024

Tagrisso is at the center of AstraZeneca’s oncology resurgence. The drug, which first won approval in the U.S. in 2015, is the company’s second highest selling product, after the diabetes and kidney disease treatment Farxiga. It has become a go-to […]

February 20, 2024

On Feb. 16, 2024, FDA announced the approval of Iovance Biotherapeutics’ Amtagvi (lifileucel), which the agency said in a press release is the first cellular therapy indicated for treating adult patients with unresectable or metastatic melanoma that has been previously […]

February 19, 2024

The US regulator has approved the drug after a priority review on the strength of data from the FLAURA 2 study showing that the combination of EGFR inhibitor Tagrisso (osimertinib) and chemo cut the risk of disease progression or death […]

February 15, 2024

The Cambridge, Massachusetts-based company is offering 19.2 million shares at $6.25 each and has scaled up its earlier expectations for the proceeds from $125 million. Prime Medicine is working on a pipeline of 18 programmes aimed at correcting pathogenic mutations […]

February 13, 2024

Both companies have entered into a license and option agreement, as well as a securities purchase agreement, in connection with the collaboration. Under the terms of the agreement, BioNTech will purchase $200m of Autolus’ US depositary shares in a private […]

February 12, 2024

Researchers from the Massachusetts Institute of Technology (MIT) and the Singapore-MIT Alliance for Research and Technology have developed a plastic microfluidic chip to improve the safety and effectiveness of cell therapy treatments for patients living with spinal cord injuries. In […]

February 12, 2024

The fast-track designation from the US regulator recognises that bepirovirsen is the only single agent in phase 3 that has shown the potential to achieve a “functional cure” when combined with standard CHB therapies based on oral nucleoside or nucleotide […]