Regenerative Medicine

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.

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

Analysis: Syncona unveils strategy after merger

Syncona Investment Management Ltd, an investment company in which the Wellcome Trust has a significant holding, reported total net assets of £895 million at the end of its fiscal year on 31 March, up from £472 million a year earlier. This follows its merger in November 2016 with BACIT Ltd, an investor in diversified portfolios based in the Guernsey Islands.

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Location

United Kingdom

Shire advances gene therapy

Shire Plc is advancing a candidate gene therapy acquired from its 2016 purchase of Baxalta, with plans to take the product into a first human trial in patients with haemophilia A. The product is a factor VIII gene therapy delivered in an adeno-associated virus (AAV) vector.

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Location

Ireland

TiGenix readies for US expansion

TiGenix has joined the growing number of European companies with a presence in the US to open an office in Cambridge, Massachusetts and prepare for the registration of its lead stem cell therapy product intended to treat fistulas in patients with Crohn’s disease.

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Location

Belgium

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