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Caraway Therapeutics

- PMLiVE

Merck agrees on $2.2bn deal with Quotient Therapeutics to develop IBD treatment

Quotient’s somatic genomics platform technology will be used to develop the treatment

- PMLiVE

UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

- PMLiVE

Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

- PMLiVE

Moderna and Merck announce data from melanoma combination therapy study

Skin cancer is one of the most common cancers in the US, with melanoma accounting for the majority of skin cancer deaths

- PMLiVE

Merck announces agreement with US government to reduce prices of prescription medication

The pharma company will invest more than $70bn in the US to boost production and innovation

- PMLiVE

Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

- PMLiVE

FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

- PMLiVE

AstraZeneca announces $2bn investment in US manufacturing

The new and expanded facilities will support production of rare disease drug treatments

- PMLiVE

Merck agrees deal with Cidara Therapeutics worth almost $9.2bn

Cidara’s long-acting viral flu drug showed significant protection in phase 2 results

- PMLiVE

AstraZeneca reports positive results from rare disease trial

HES is a group of rare disorders characterised by persistently elevated levels of white blood cells

- PMLiVE

Merck ends development agreement with Dr Falk Pharma for monoclonal antibody

Merck will take full control of developing MK-8690, an investigational monoclonal antibody candidate

- PMLiVE

Merck and Blackstone agree on funding for cancer protein antibody

The funding will support late-stage clinical development of the investigational antibody-drug conjugate

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