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The authorisation is based on the data from the global, open label FOENIX-CCA2 trial. It enrolled a total of 103 adult patients from the Netherlands, UK, Spain, Italy,

A single-time, recombinant adeno-associated virus (rAAV)-mediated gene therapy for ocular disease, SKG0106 is administered intravitreally. The global Phase I trial will begin shortly. SKG0106 consists of a new AAV

The treatment is indicated for usage in areas that are not covered by the reference medicine Humira’s regulatory exclusivity. The indications include inflammatory ailments namely juvenile idiopathic arthritis,

Intended to treat adult patients, fidanacogene elaparvovec is an investigational therapy that comprises a bio-engineered adeno-associated virus (AAV) capsid and a human coagulation Factor IX (FIX) gene variant.

An oral inhibitor of hypoxia-inducible factor prolyl hydroxylase, daprodustat is intended for usage in adult patients who are on chronic maintenance dialysis. The company is developing the therapy

Former investors such as Matrix Capital Management Company and Alexandria Venture Investments, who took part in the firm’s Series A round, joined the latest fundraise along with new

Bluebird is seeking approval for treating a rare genetic disease, SCD, in patients aged 12 years and above having a history of vaso-occlusive events. A modified form of

The NDA was accepted by the regulatory agency for review with a PDUFA target action date set for 17 June 2023. As per the CRL, the regulatory body

With the acquisition, Rosemont will expand its existing portfolio and gain access to the new pipeline of products, providing lifesaving pharmaceuticals across the UK and within overseas markets.