genetic diseases

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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.

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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.

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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.

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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

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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.

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Rachel McMinn, Neurogene's founder and chief executive

Neurogene shares fall on Rett syndrome gene therapy data

Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share pric

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

Neurogene shares fall on Rett syndrome gene therapy data

LSX2026: Addressing adult eye cancer, with Roi Raz

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genetic diseases

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