Regenerative Medicine

Second gene therapy approved

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.

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Plasticell and GSK in stem cell collaboration

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.

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FDA committee recommends Spark’s gene therapy

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.

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Location

United States

Meeting Report: Lessons from the first ATMPs

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.

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Location

Germany

Gene therapy trial featured

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.

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Location

United States

FDA’s Gottlieb announces policy on stem cells

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.

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Location

United States

Gilead to acquire Kite for $11.9 billion

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.

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Location

United States

Embryo edits solve some problems, unleash new ones

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.

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Location

United States

Nightstar recruits executive after Series C

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.

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Location

United Kingdom

FDA designation for Spark

A gene therapy developed by Spark Therapeutics Inc for an inherited retinal disease has received a paediatric designation from the US Food and Drug Administration. The decision is the agency’s way of highlighting the treatment’s potential for children and young adults.

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Location

United States

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