Rare diseases

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AlveoGene debuts to develop inhaled gene therapy technology

Today saw the debut of a new UK-based biotech, called AlveoGene, which has been formed to develop therapies based on a lentiviral vector platform used to deliver gene ther

News

Moderna looks to its post-COVID growth prospects

With sales of its COVID-19 vaccines on the wane, Moderna has showcased the projects it will look to for the next phase of its development, headed by new vaccines for influ

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Clinigen launches scheme to support rare disease patients

Clinigen has launched a new programme in the UK that aims to help organisations representing patients with rare diseases participate in R&D for new medicines and suppo

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One year on, ABPI asks why IMF hasn't funded any drugs

The Innovative Medicines Fund (IMF), set up in the UK last year to fast-track NHS access to promising new drugs, still hasn't provided any funding for a new medicine, and

News

Ipsen drug is first therapy for rare bone disease FOP in US

With FDA approval, Ipsen's marathon effort to bring palovarotene to market for the rare disease fibrodysplasia ossificans progressiva (FOP) has finally crossed the finish

News

Chiesi partners Aliada on brain-penetrating CNS therapies

Rare disease specialist Chiesi Pharma has forged an alliance with US biotech Aliada Therapeutics that will apply a technology designed to get drug molecules across the blo

AlveoGene debuts to develop inhaled gene therapy technology

Moderna looks to its post-COVID growth prospects

Clinigen launches scheme to support rare disease patients

One year on, ABPI asks why IMF hasn't funded any drugs

Ipsen drug is first therapy for rare bone disease FOP in US

Chiesi partners Aliada on brain-penetrating CNS therapies

CAR-TCR 2023 – Jason Noon

Turning drug development on its head

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