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Lilly and MeiraGTx partner in $475m ophthalmology deal

The companies will develop and commercialise therapies for genetic conditions

The (clinical) trials and tribulations of gene therapy – why meaningful patient engagement strategies matter

By Sarah Mohamad and Lauren Coleman-Bennett

Amiculum

What do people look for in gene therapy materials? Results from our survey of patients and relatives

By Sam Fraser

Amiculum

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CSL Behring announces four-year results for haemophilia B gene therapy Hemgenix

The genetic bleeding disorder affects an estimated one per every 19,283 male births in the US

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NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease

About 15,000 people in England are living with the inherited blood disorder

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Vertex and Orum partner on gene editing conditioning drugs in deal worth $945m

The agreement gives Vertex the rights to use Orum’s targeted protein degradation technology

regeneron headquarters

Regeneron and Mammoth announce gene editing partnership worth $370m per target

The companies will aim to develop CRISPR-based therapies for multiple tissues and cell types

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FDA publishes final industry guidance for CAR-T and gene therapies

The new guidance can also be applied to other genetically modified lymphocyte products, including  CAR NK cells

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Study identifies hundreds of priority targets for cancer drug discovery

Researchers identified 370 priority drug candidates across 27 types of cancer

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AstraZeneca and Cellectis enter gene therapy partnership worth over $2.2bn

The deal gives AZ access to Cellectis’ gene editing technologies and manufacturing capabilities

Biomarin

BioMarin announces first patient dosed with haemophilia A gene therapy in Europe

The therapy was granted conditional marketing authorisation by the EC in August 2022

regeneron headquarters

Regeneron to acquire Decibel Therapeutics in deal worth up to $213m

The transaction expands the company’s gene therapy and hearing loss pipeline

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