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CHAPLE disease

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LEO Pharma to acquire Replay gene therapy platform

The acquisition will advance innovation for patients with rare skin diseases

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Shionogi starts phase 2 clinical trial for Pompe Disease

The rare disease affects around one in every 22,000 people worldwide

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PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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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

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Sanofi announces positive results for venglustat in type 3 Gaucher disease

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

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Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

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FDA approves first gene therapy for Wiskott-Aldrich syndrome

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

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AstraZeneca announces $2bn investment in US manufacturing

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

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AstraZeneca reports positive results from rare disease trial

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

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Regeneron’s Lynozyfic granted FDA accelerated approval to treat multiple myeloma

More than 36,000 new cases of the blood cancer are expected to be diagnosed in the US this year

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FDA unveils new voucher programme to accelerate drug review process

The programme is designed to reduce the review time for companies supporting US national interests

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Regeneron gains rights to Hansoh’s GLP-1/GIP receptor agonist in deal worth $2bn

The candidate is being evaluated in a phase 3 obesity trial and phase 2b diabetes study

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