Regenerative Medicine

Synpromics works with UCL

Synpromics Ltd is bringing its technology for creating synthetic promoters into a partnership with a unit of University College London (UCL) in order to help researchers there develop gene therapies for blood-based disorders. The agreement was announced on 25 January.

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Location

United Kingdom

New delivery for gene therapy

The prospect of being able to re-dose a gene therapy has come into the fore with the launch on 4 January of Generation Bio Inc, a company with technology for delivering DNA directly to the liver using lipid nanoparticles rather than a viral vector.

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Location

United States

Takeda to acquire cell therapy company TiGenix

Japan’s Takeda Pharmaceutical Company Ltd has announced plans to acquire the Belgium-based cell therapy company TiGenix NV for €520 million, a company with which it already has a licensing deal for the company’s lead product.

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Location

Belgium

Spark offers outcomes-based rebates for gene therapy

Spark Therapeutics Inc has set the price per patient of its new gene therapy for retinal dystrophy at $850,000 but at the same time opened negotiations with leading US insurers to link payments with the success of the treatment.

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Location

United States

TiGenix tightens allogeneic focus

Following a positive opinion in Europe for its cell therapy for fistulas in Crohn’s disease, TiGenix NV has tightened its therapeutic focus to concentrate on allogeneic therapies and in particular, on inflammatory conditions associated with immune-mediated diseases.

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Location

Belgium

Investors support Orchard Therapeutics’ gene therapies

Orchard Therapeutics Ltd has completed an oversubscribed $110 million Series B financing round to support its pipeline of gene therapies which is led by a clinical-stage product for the inherited disorder, adenosine deaminase severe combined immunodeficiency (ADA-SCID).

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FDA approves new gene therapy

The first gene therapy for patients with an inherited retinal disease was authorised by the US Food and Drug Administration on 19 December, bringing the number of approved gene therapies in the US up to three. Luxturna (voretigene neparvovec-rzyl), was developed by Spark Therapeutics Inc.

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Location

United States

Positive opinion for stem cell therapy

The European Medicines Agency has issued a positive opinion for an allogeneic stem cell therapy to treat complex perianal fistulas in patients with Crohn’s disease. The product, Alofisel (darvadstrocel), was developed by TiGenix NV of Belgium.

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Location

Belgium

Orchard Therapeutics partners with Généthon

Orchard Therapeutics Ltd has obtained rights to technology developed by the French research institution Généthon to continue development of a new gene therapy for the immune deficiency, X-linked chronic granulomatous disease.

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Location

United Kingdom

New collaboration on gamma delta T cells

Bluebird bio Inc has teamed up with the Edinburgh, UK-based TC BioPharm Ltd to create new immunotherapies for cancer based on gamma delta T cells, a small subset of T cells involved in the initiation and propagation of immune responses.

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Location

United Kingdom

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