The novel gene therapy is being developed for the lysosomal storage disease Sanfilippo syndrome type A (MPS IIIA)
News
Roche buys rights to Sarepta’s DMD gene therapy
Sarepta will receive $1.15 billion upfront and an equity investment
NHS fast-tracks access to Epidyolex for severe epilepsy
Doctors will be able to prescribe the drug from January 6
US approves Merck’s Ebola vaccine
The approval marks a “significant milestone in Ebola prepardness and prevention”
ViiV’s HIV regimen hit with setback in the US
US regulators have declined ViiV Healthcare’s application to market an investigational long-acting regimen for the treatment of HIV
AZ divests rights to Arimidex and Casodex
Juvisé has paid upfront $181 million to AZ for the medicines
INTCR 2020 entry deadline fast approaching
The 2020 PharmaTimes International Clinical Researcher of the Year competition will be closing for entry on the 23rd January 2020.
Roche recruits Rheos to explore immunometabolism
Rheos will receive an upfront cash payment of $42.5 million, with up to $90 million available for specified research and preclinical development milestones.
Fampyra recommended in Wales after positive results in MS
The decision means that Wales is the first country in the UK to recommend funding for the drug, after the SMC refused it.
Janssen depression drug given go-ahead in Europe
Major depressive disorder affects approximately 40 million people across Europe and 1.8 million adults in England alone.
Rinvoq gains EU approval for rheumatoid arthritis
The decision is based on results from the Phase III SELECT studies.
First patient treated with ‘acoustic cluster therapy’
The new technology uses microscopic clusters of bubbles and liquid droplets to enhance the delivery of chemotherapy drugs to tumours.
Royal College urges PM to reconsider review of ‘sin taxes’
According to HMRC, sugar tax receipts for 2019 were £251 million, representing an increase of around £100 million from 2018.
Scotland gives Roche’s haemophilia A drug the go-ahead
The drug can be self-administered by injection under the skin at multiple dosing options.
Roche’s Kadcyla cleared in Europe for certain breast cancers
On the news, Kadcyla in this setting is now approved in 27 countries worldwide.
