Cell and Gene Therapy, Industry
March 26, 2024
Via: PharmaphorumThe 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 […]
Cell and Gene Therapy, Industry
March 20, 2024
Via: Biopharma DiveGene 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 […]
Cell and Gene Therapy, Clinical Trials, Industry, Research and Development
March 6, 2024
Via: PMLiVEAffecting 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 […]
February 27, 2024
Via: Biopharma DiveThe FDA’s decision sets back many years of effort by Minerva to secure approval of its drug for schizophrenia, called roluperidone and designed to treat so-called negative symptoms of the condition. The company, which licensed rights to roluperidone from Mitsubishi […]
Cell and Gene Therapy, Industry
February 23, 2024
Via: Biopharma DiveRoctavian’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
February 23, 2024
Via: PMLiVEThe 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
February 21, 2024
Via: Biopharma DiveA 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
February 20, 2024
Via: Biopharma DiveTagrisso 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 […]
Clinical Trials, Research and Development
February 20, 2024
Via: Biopharma DiveAnalysts have followed Rapt as they view its drug, an oral pill dubbed zelnecirnon, as a possible competitor to Dupixent in conditions like eczema. Dupixent, which was developed by Sanofi and Regeneron, is a multibillion-dollar product approved for a range […]
Cell and Gene Therapy, Industry
February 20, 2024
Via: Biopharm InternationalOn 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 20, 2024
Via: PharmaphorumIf approved, Vyvgart Hytrulo (efgartigimod alfa) will be the first drug in the FcRn blocker class to treat CIDP, a rare autoimmune disease of the peripheral nervous system that causes fatigue, muscle weakness, and loss of feeling that can lead […]
Cell and Gene Therapy, Industry
February 19, 2024
Via: PharmaphorumThe 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 14, 2024
Via: Biopharma DivePancreatic cancer remains one of the most difficult tumors to treat, with few good options after cancerous cells have spread more widely. More than 60,000 people are diagnosed with the adenocarcinoma form each year, according to Ipsen, often after metastases. […]
Cell and Gene Therapy, Industry
February 13, 2024
Via: PMLiVEBoth 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
February 12, 2024
Via: PharmaphorumThe 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
February 8, 2024
Via: Biopharma DiveCRISPR 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
February 7, 2024
Via: Biopharma DiveAstraZeneca 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
Via: Biopharm InternationalBiovian, a contract development and manufacturing organization (CDMO), announced on Feb. 1, 2024 that it was combining with CDMO 3P Biopharmaceuticals to form 3PBIOVIAN under the two companies’ common shareholder, Keensight Capital. The new group, 3PBIOVIAN, will offer end-to-end development […]
Cell and Gene Therapy, Industry
February 5, 2024
Via: PharmaphorumOn 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 2, 2024
Via: PharmaphorumIf the biologics license application (BLA) is approved, afami-cel will become the first engineered T-cell therapy for solid tumours and the first effective treatment option for advanced synovial sarcoma in more than a decade, according to the Anglo-American company. Synovial […]