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Category: Cell and Gene Therapy


Cell and Gene Therapy, Industry

BioMarin preaches patience amid slow sales for hemophilia gene therapy

February 23, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

Johnson & Johnson’s Tecvayli receives FDA approval for reduced dosing in multiple myeloma patients

February 23, 2024

Via: PMLiVE

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 […]


Cell and Gene Therapy, Industry

NEJM paper fills in details on ‘remarkable’ CAR-T result in autoimmune disease

February 21, 2024

Via: Biopharma Dive

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, […]


Cell and Gene Therapy, Industry

AstraZeneca gets new Tagrisso OK as drug succeeds in another trial

February 20, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

FDA Approves Amtagvi, First Cell Therapy for Skin Cancer

February 20, 2024

Via: Biopharm International

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 […]


Cell and Gene Therapy, Industry

Tagrisso-chemo cleared in US as 1st-line lung cancer therapy

February 19, 2024

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry

Gene-editing firm Prime Medicine files $140m public offer

February 15, 2024

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry

BioNTech and Autolus enter CAR-T cell therapy collaboration worth over $250m

February 13, 2024

Via: PMLiVE

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 […]


Cell and Gene Therapy, Industry

Scientists develop micofluidic chip to make cell therapy safer

February 12, 2024

Via: Pharma Times

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 […]


Cell and Gene Therapy, Industry

FDA fast-tracks GSK’s chronic hep B therapy bepirovirsen

February 12, 2024

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry

Gene editing biotech Metagenomi pulls off nearly $94M IPO

February 8, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

AstraZeneca to invest $300M in US cell therapy plant

February 7, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

4DMT gene therapy cuts need for Eylea in wet AMD trial

February 5, 2024

Via: Pharmaphorum

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 […]


Cell and Gene Therapy, Industry

Regeneron Forms R&D Cell Therapy Unit from 2seventy bio Platform Acquisition

February 1, 2024

Via: Biopharm International

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 […]


Cell and Gene Therapy, Industry

FDA Issues Guidance on CAR-T Cell Product Development

February 1, 2024

Via: Biopharm International

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 […]


Cell and Gene Therapy, Industry

FDA Give Guidance on Human Genome Editing in Gene Therapies

January 31, 2024

Via: Biopharm International

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 […]


Cell and Gene Therapy, Industry

CAR-T for lupus: the ‘tip of the iceberg’ for cell therapy in autoimmune disease

January 30, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

Regeneron to acquire 2seventy’s cell therapy pipeline

January 30, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

AstraZeneca, Daiichi aim for first pan-tumor ADC approval

January 29, 2024

Via: Biopharma Dive

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 […]


Cell and Gene Therapy, Industry

EMA recommends earlier use of Bristol Myers cell therapy for multiple myeloma

January 26, 2024

Via: Biopharma Dive

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 […]