Semma Therapeutics Inc, a company founded by the Harvard University Professor Douglas Melton, has raised $114 million in an oversubscribed Series B financing round to bring a stem cell therapy for Type1 diabetes into the clinic.
The US Food and Drug Administration has approved a second gene therapy to treat cancer, giving a further boost to developers of the new technologies. The approval is for Yescarta (axicabtagene ciloleucel), a chimeric antigen receptor (CAR) T cell therapy to treat adults with certain types of large B-cell lymphoma.
GlaxoSmithKline Plc, which has a gene therapy on the market, is broadening its portfolio of advanced therapy medicinal products to include potential new treatments derived from induced pluripotent stem cells (iPSC). iPSCs are a type of pluripotent stem cell that can be generated directly from adult cells.
Spark Therapeutics Inc’s gene therapy for the treatment of inherited retinal dystrophy has received the unanimous backing of an advisory committee of the US Food and Drug Administration ahead of the agency’s decision on marketing in early 2018.
It is one thing to research and develop an advanced therapy medicinal product (ATMP), but quite another to bring it successfully to market. Among the nine ATMPs approved in Europe since 2009, only five are still on the market.
A Phase 3 trial of a candidate gene therapy for retinal disorder has been highlighted in The Lancet as a possible standard for future trials of genetic medicines. The trial was conducted by Spark Therapeutics Inc whose therapy, voretigene neparvovec, is under review at the US Food and Drug Administration.
The US Food and Drug Administration has taken steps to curb the activities of unlicensed stem cell clinics while preparing for the roll-out in the autumn of a new regulatory framework for developers of cell and tissue-engineered products.
Gilead Sciences Inc has made a dramatic entry to the field of regenerative medicine with an agreed bid for Kite Pharma Inc which has a portfolio of engineered cell therapies, the most advanced of which is under review in both the US and the EU.
An experiment in which CRISPR-Cas9 was used to repair a gene mutation in a human embryo appears to have solved two safety problems associated with this technology while raising ethical questions about its long-term application.
UK-based Nightstar Ltd has recruited David Lubner to its board of directors less than a month after raising $45 million in a Series C financing round to advance its lead gene therapy product for choroideremia, an inherited cause of progressive blindness.