Cell & Gene Therapies

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EMA pilot to provide regulatory support to selected ATMP developers

By Rachel Arthur

The European Medicines Agency (EMA) will provide up to five selected advanced therapy medicinal products (ATMPs) with enhanced regulatory support - and in the process hopes to learn how to improve the regulatory processes for other ATMP drugs in the future.

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Rocket Pharmaceuticals to acquire Renovacor

By Rachel Arthur

Rocket Pharmaceuticals, a late-stage, clinical biotech advancing a pipeline of genetic therapies for rare childhood disorders, will acquire Renovacor: helping it strengthen its position in AAV-based cardiac gene therapy.

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bluebird bio gets green light for Skysona gene therapy in the US

By Rachel Arthur

The US Food and Drug Administration (FDA) has granted Accelerated Approval for bluebird bio’s Skysona (elivaldogene autotemcel), also known as eli-cel, to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral...

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Vertex to acquire ViaCyte, targeting type 1 diabetes

By Rachel Arthur

Vertex Pharmaceuticals will acquire ViaCyte, a privately held biotech focused on delivering novel stem cell-derived cell replacement therapies as a functional cure for type 1 diabetes (T1D), for $320m.

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Kite secures third approved cell therapy indication in Europe

By Jane Byrne

The European Commission (EC) has approved Kite’s CAR T-cell therapy, Yescarta, (axicabtagene ciloleucel) for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy.

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Matica Bio opens new Texas cell and gene therapy facility

By Rachel Arthur

CDMO Matica Bio has opened a new 45,000-square-foot facility dedicated to the production of viral vectors and cell-based products used in cell and gene therapies, vaccines, oncolytic therapies and other genetic medicines.

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Ocugen starts clinical trials for retinal gene therapy candidate

By Rachel Arthur

The first patient has been dosed in the Phase 1/2 clinical trial of OCU400, a modifier gene therapy candidate for the treatment of retinitis pigmentosa (RP) resulting from mutations in the nuclear receptor subfamily 2 group E member 3 (NR2E3) and Rhodopsin...

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