Category: Cell and Gene Therapy

February 8, 2024

CRISPR gene editing captured headlines in recent months with the landmark approval of Casgevy, the first medicine made from the Nobel Prize-winning technology. But that milestone was only part of a larger story, of which an emerging generation of drugmakers […]

February 7, 2024

AstraZeneca is ramping up in cell therapy after a late entrance to the field. In 2022, it made its first significant acquisition in the area, buying up privately held Neogene Therapeutics. And the follow year, it formed a partnership with […]

February 5, 2024

On the strength of the results, 4DMT is planning to start a phase 3 trial of the gene therapy by the end of the first quarter of 2025. The data from the phase 2 PRISM study comes from a cohort […]

February 1, 2024

FDA published final guidance on Jan. 30, 2024 that provides consideration for the development of chimeric antigen receptor (CAR) T-cell products. According to FDA, “[CAR T-cell] products are human gene therapy products in which the T cell specificity is genetically […]

February 1, 2024

On Jan. 30, 2024, Regeneron Pharmaceuticals (Regeneron) announced the formation of a new R&D unit, Regeneron Cell Medicines, founded on an agreement with 2seventy bio, a US-based immuno-oncology cell therapy company. Under the agreement, Regeneron acquires full development and commercialization […]

January 31, 2024

FDA published a final guidance document, Human Gene Therapy Products Incorporating Human Genome Editing, on Jan. 30, 2024. The document provides recommendations for developing gene therapy products incorporating genome editing (GE) of human somatic cells. Recommendations include information needed in […]

January 30, 2024

The clue came, as they often do, from an unexpected source. Some sixteen months ago, a small study of five people in Germany pointed to a new direction for the high-profile field of cell therapy. The study showed a cellular […]

January 30, 2024

Regeneron Pharmaceuticals is expanding its cell therapy research, announcing a deal Tuesday to acquire the drug pipeline of 2seventy Bio and bring on many of the smaller biotechnology company’s employees. The experimental cell therapies Regeneron acquires will be housed in […]

January 29, 2024

The Food and Drug Administration could soon decide on whether to make AstraZeneca and Daiichi Sankyo’s drug Enhertu available for any solid tumor with a specific genetic signature. The partners on Monday said the FDA is reviewing their request to […]

January 26, 2024

If cleared by the European Commission, Abecma would be available for third-line use in the EU’s 27 member countries, a needed boost for a medicine that’s losing ground to Johnson & Johnson’s rival therapy Carvykti. Both medicines are part of […]

January 25, 2024

Five of six Chinese children born deaf due to a rare genetic defect now have the ability to hear, thanks to an experimental gene therapy. The therapy involved a hollowed-out virus loaded with a healthy version of the gene responsible […]

January 24, 2024

An 11-year-old boy who was born deaf can hear after receiving an experimental gene therapy, developer Eli Lilly reported on Tuesday. The boy, identified as Aissam Dam by The New York Times, was the first participant treated in a small […]

January 23, 2024

The CAR-T therapies — Abecma and Breyanzi from Bristol Myers Squibb, Carvykti from Johnson & Johnson and Legend Biotech, Kymriah from Novartis, and Tecartus and Yescarta from Gilead — can drive deep and durable responses in patients with lymphoma, leukemia […]

January 18, 2024

Jaguar Gene Therapy is spinning out a new company to handle the specialized work of manufacturing cell and gene therapies, launching Wednesday a wholly owned subsidiary called Advanced Medicine Partners. The spinout, which is backed by investors Deerfield Management, Arch […]

January 17, 2024

Casgevy (exagamglogene autotemcel or exa-cel) became the first drug based on the CRISPR/Cas9 gene-editing technology to be approved towards the end of last year, when it was cleared by the FDA for sickle cell disease (SCD). The UK was the […]

January 12, 2024

The new GenePHIT study is enrolling patients with non-ischaemic cardiomyopathy and New York Heart Association (NYHA) class III heart failure who have been medically stable for at least 4 weeks. People with this stage of CHF will have a marked […]

January 4, 2024

Germany’s Merck KGaA is paying $45 million upfront for rights to a potentially first-in-class drug for colorectal cancer (CRC) being developed by New York biotech Inspirna. The agreement gives Merck exclusive ex-US rights to ompenaclid – an oral inhibitor of […]

January 3, 2024

Hemophilia and other blood disorders like sickle cell disease and beta thalassemia have drawn intense investment from genetic medicine developers. in December, the FDA approved two such treatments for sickle cell, clearing Vertex Pharmaceuticals’ gene editing drug Casgevy and Bluebird […]

January 2, 2024

Novartis is a leading gene therapy developer. In 2019, its SMA treatment Zolgensma became one of the first genetic medicines cleared for use in the U.S., and so far has been the field’s most lucrative product. Since then, gene therapy […]

December 29, 2023

Replay, a genome writing company, announced that it has entered into separate agreements with the National Institutes of Health (NIH) and Miltenyi Biotec, a provider of products and services for biomedical research and cellular therapy, that will lead to the […]