We were pleased to announce back in August 2020 that the first person was dosed with ZF874, a potential treatment for alpha-1-antitrypsin deficiency. Then on the 24th September we published the details for HMR who are running the trail on behalf of Z Factor, asking for PiMZ people to help.
15th October 2020 Vertex Pharmaceuticals announced that based on early data of its Phase II trial of VX-814 in Alpha-1 antitrypsin deficiency (AATD), it is halting the trial and ending development of the drug. AATD is a genetic disease resulting in a missing liver protein. Children with AATD either do not produce
If you are a healthy PiMZ then please consider taking part in this exciting Alpha-1 clinical trial. Please contact HMR direct using the contact email shown below quoting 20B005 in your email. If you have any concerns with participating right now due to COVID, HMR state private transport can be
Z Factor Ltd, a Cambridge-based drug development company spun out of the University of Cambridge, reveals that the first human volunteer was dosed today with ZF874, its novel treatment for alpha-1-antitrypsin deficiency (AATD). AATD is a common genetic disorder, affecting around in 1 in 2000 people in Western countries, where
Z Factor Ltd, a Cambridge-based drug development company spun out of the University of Cambridge, announced that the first human volunteer was dosed today with ZF874, its novel treatment for alpha-1-antitrypsin deficiency (AATD). AATD is a common genetic disorder, affecting around in 1 in 2000 people in Western countries, where
RNA interference (RNAi), sometimes called gene silencing, is an approach to therapeutics in certain diseases that are caused by the production of abnormal proteins or an overproduction of proteins, which can be treated by turning off or silencing the gene. One of the companies working on the cutting edge of
LEXINGTON, Mass.–(BUSINESS WIRE)–Dec. 17, 2019– Dicerna™ Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that the European Commission (EC) has granted orphan drug designation to Dicerna’s DCR-A1AT for the treatment of congenital alpha-1 antitrypsin (A1AT) deficiency based on a positive opinion from the
Rehovot, Israel, December 16, 2019 — Kamada Ltd. (NASDAQ & TASE: KMDA), a plasma-derived protein therapeutics company, announced today that the first patient has been randomized in Europe into its pivotal Phase 3 InnovAATe clinical trial evaluating the safety and efficacy of the Company’s proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy for