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 […]
Cell and Gene Therapy, Industry
March 1, 2024
Via: Biopharm InternationalBioCina, 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 […]
Cell and Gene Therapy, Industry
February 29, 2024
Via: Biopharma DiveKenai 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 […]
Cell and Gene Therapy, Industry
February 27, 2024
Via: World Pharma NewsBayer 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, […]
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 […]
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 […]
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 […]
Cell and Gene Therapy, Industry
February 15, 2024
Via: PharmaphorumThe 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
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: Pharma TimesResearchers 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
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 […]
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 […]
Cell and Gene Therapy, Industry
February 1, 2024
Via: Biopharm InternationalOn 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
February 1, 2024
Via: Biopharm InternationalFDA 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
January 31, 2024
Via: Biopharm InternationalFDA 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 […]