genetic diseases

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PTC Therapeutics' chief executive Matthew Klein.

Germany will be first launch for PTC's phenylketonuria drug

PTC Therapeutics' treatment for the rare disease PKU, Sephience, will launch in its first market next month after getting EU approval.

News

KJ's parents, Kyle and Nicole, and his three siblings

World first as infant gets personalised CRISPR therapy

In a medical breakthrough, a child diagnosed with a rare genetic disorder has received a bespoke CRISPR gene editing therapy to correct the mutation.

News

Beam launches $500m financing on base-editing trial data

Beam says its base-editing drug has met its goals in a phase 1/2 trial in genetic disease AATD, and it wants to raise $500m to take it forward.

News

Biogen bags rights to epilepsy drug from Stoke in $550m deal

Biogen has bolstered its pipeline by licensing ex-US rights to a drug for Dravet syndrome – a rare form of epilepsy – from Stoke Therapeutics.

News

Neurogene drops high-dose arm in Rett study after death

The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died

News

Neurogene falls again on Rett gene therapy side effects

Neurogene has halted patient enrolment in the high-dose arm of its trial of Rett syndrome gene therapy, sparking another run on its shares.

Germany will be first launch for PTC's phenylketonuria drug

World first as infant gets personalised CRISPR therapy

Beam launches $500m financing on base-editing trial data

Biogen bags rights to epilepsy drug from Stoke in $550m deal

Neurogene drops high-dose arm in Rett study after death

Neurogene falls again on Rett gene therapy side effects

‘Insulin for muscle’ and the AI platform behind it, with Han...

The value of a partner, globally and locally

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