Cell and gene therapy developers need staff with skills according to McKinsey & Company, which says making such therapies is laborious, complex, and expensive and few firms are ready to take on these challenges. The management consulting group wrote about the burgeoning cell and gene therapy industry in a blog post this month, writing that as more cell and gene therapies come to market industry will need to invest in skills. “Five years ago, these treatments were really just ideas…
The European Medicines Agency (EMA) endorsement of BioMarin’s hemophilia A gene therapy comes days after the US recommended two bluebird bio gene therapy products. The Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Roctavian (valoctocogene roxaparvovec), laying the ground for the approval of the first gene therapy for the treatment of hemophilia A. Roctavian is based on an adeno-associated virus (AAV) containing the gene for factor VIII, which, once administered as a one-off therapy,…
Small molecule biotech Galapagos has jumped into the cell therapy space through the acquisitions of Cellpoint and AboundBio for €125 ($130) million and $14 million, respectively. The deals move Galapagos into the advanced therapy space, through the addition of Cellpoint’s chimeric antigen receptor (CAR) T-cell therapy pipeline and decentralized manufacturing technology. Dutch biotech CellPoint’s point-of-care supply model, which leverages with Lonza’s closed, automated Cocoon platform, claims to offer 7-day delivery of CAR-T therapies while avoiding logistical complexities associated with the…
Cell therapy developers must think carefully before adopting a decentralized manufacturing model say researchers, who suggest the benefits of bedside production are outweighed by technical challenges. The advice, presented in a study in the Journal of Translational Medicine, is based on an assessment of current cell therapy production models which found that the decentralized, multi-production site approach is gaining in popularity, particularly in trials. “For cellular therapy clinical trials involving multiple sites some people are advocating a decentralized model of…
An FDA Advisory Committee has unanimously agreed bluebird bio’s gene therapy elivaldogene autotemcel (eli-cel) outweigh the risks in treating early active cerebral adrenoleukodystrophy (CALD). The US Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) met yesterday to evaluate bluebird bio’s eli-cel as a treatment of early active cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC)…
Investment and innovation will continue to pour into autologous CAR-T therapies even as allogeneic products begin to impact the market, according to Gilead Sciences. Gilead Sciences is a pioneer in the cell therapy space, acquiring Kite Pharma in 2017 months ahead of the FDA approving Yescarta (axicabtagene ciloleucel), the second CAR-T therapy to achieve commercial success. Gilead won approval for a second autologous CAR-T, Tecartus (brexucabtagene autoleucel), in July 2020. Talking at the Bernstein Annual Strategic Decisions Conference last week,…
Samsung Biologics has produced the first batch of drug substance for GreenLight Biosciences from its newly built mRNA facility in Korea. In June 2021, Korean contract development and manufacturing organization (CDMO) Samsung Biologics announced its intentions to enter the messenger RNA (mRNA) space through the building of a vaccine drug substance production facility at its biomanufacturing campus in Songdo, Incheon. Five months later, and the firm won a contract to make Boston-based biotech firm GreenLight’s messenger RNA COVID-19 vaccine candidate…
First-generation CAR-Ts are “fantastic” says AbbVie, but administrative difficulties mean bispecific antibodies are more likely to provide earlier lines of treatment in conditions like blood cancers. This week, an Evaluate Pharma report touted AbbVie to become the biggest pharma company by sales in 2028, pulling in a predicted $65.7 billion, despite the expected drop in sales of its blockbuster Humira (adalimumab). But one modality that is unlikely to play significantly in the firm’s topline is chimeric antigen receptor (CAR) T-cell…
Evonik’s lipid manufacturing facility in Lafayette, Indiana will service demand from the Biomedical Advanced Research and Development Authority (BARDA) for mRNA-based therapies beyond COVID-19 vaccines. Evonik’s second largest site in the US will benefit from a lipid manufacturing facility. Construction is set to begin in early 2023, with the plant expected to be operational by 2025. Of the $220 million being invested in Tippecanoe, $150 million will come from the US Department of Health and Human Services (HHS) division BARDA…
Regeneron has agreed to supply Elicio Therapeutics with the drug Libtayo for clinical trials of its candidate cancer vaccine, ELI-002. The trial will examine the products as a combination in patients with KRAS (Kirsten rat sarcoma virus)-driven tumors including stage III and IV non-small cell lung cancer, stage IV colorectal cancer and unresectable, locally advanced or oligometastatic pancreatic ductal adenocarcinoma. KRAS mutations are among the most prevalent human cancers, driving 32% of lung cancers, 40% of colorectal cancers and 85%…