Meeting Report: cell and gene therapy readouts in 2015

Written by Victoria English

Scientists expect to have readouts next year from cell and gene therapy trials that could show that regenerative medicine is finally gaining traction.

Scientists expect to have readouts next year from cell and gene therapy trials that could show that regenerative medicine is finally gaining traction.

This was one of the points to emerge from a meeting of regenerative medicine developers and financial industry representatives in London on 13 November. Organised by the Alliance for Regenerative Medicine, the meeting heard about late-stage development programmes at companies with prospective products in the ophthalmic, inflammatory and cardiovascular disease areas.

Leading the ophthalmology sector is Spark Therapeutics Inc of Philadelphia, Pennsylvania which has a gene therapy in Phase 3 for patients with an inherited eye disorder known as Leber’s congenital amaurosis. The treatment, which involves delivering the RPE65 gene into the back of the eye by injection, has shown a positive effect in early clinical studies. Phase 3 data is expected in the second half of next year. This will be a barometer for the industry, Soraya Bekkali, head of ophthalmology at Sanofi SA, told the meeting.

Ophthalmology has been an active space for gene therapy because many of the disorders are linked to gene mutations. The eye is also an immune privileged site and relatively easy to access. Sanofi has already in-licensed two early-stage gene therapies for rare eye disorders from Oxford BioMedica Plc. Meanwhile the Wellcome Trust subsidiary Syncona Partners recently seed funded an Oxford University spin-out called NightstaRx Ltd. Nightstar has a gene therapy in Phase 1 for an inherited eye disorder called choroideremia.

In the area of inflammatory disease, a Phase 3 read-out of an allogeneic cell therapy for fistulas in Crohn’s disease is expected in the third quarter of next year. The developer, TiGenix NV, is testing an allogeneic, adipose-derived stem cell treatment in patients with fistulas who have failed to respond to other treatments, Claudia D’Augusta told the meeting. If the results are positive, the company will make a regulatory filing in Europe shortly thereafter. TiGenix already has a cell therapy product on the market — ChondroCelect for treating cartilage defects in the knee.

One cell therapy for cardiovascular disease and another for graft versus host disease are expected to reach major thresholds in 2015. The cardiovascular product is an autologous therapy for ischaemic heart failure being developed by Cardio3 BioSciences SA using technology from the Mayo Clinic. Phase 3 data on the product, C-Cure, is expected towards the end of the year.

Meanwhile, Mesoblast Ltd of Australia has five cell therapies that are Phase 3-ready or in Phase 3. Silviu Itescu, the chief executive, said that a regulatory filing was recently made in Japan for the company’s allogeneic treatment for acute graft versus host disease. If approved, it would be the first allogeneic cell-based product to be marketed in that country. Mesoblast’s therapies are based on mesenchymal stem cells and include a candidate product for congestive heart failure.

Mr Itescu and other executives said that Japan has moved to centre stage following a decision by the government in 2013 to ease the pathway for the regulatory approval of regenerative medicines.

An additional factor is demographics. Over the next two decades the elderly population in Japan is expected to double with a rising incidence of diseases such as ischaemic stroke, said Gil van Bokkelen, CEO of Athersys Inc. Athersys has a Phase 2 cell therapy for stroke that is expected to report in the first quarter of next year. A Japanese filing is a possibility.

-Victoria English

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