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UK biotech e-therapeutics has a new CEO, with executive chairman and former Silence Therapeutics chief Ali Mortazavi chosen to spearhead the next stage in the company’s development into genesilencing and other areas. The company has considerably more cash than at the start of the year – on 31 July it had £15.1
Pharmacompanies have also made headway in rare diseases as several pipeline projects came to fruition. In summer, Alnylam’s genesilencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder Oxlumo was approved in the European Union and the US.
The present hATTR treatment market constitutes compounds that stabilize the TTR tetramers, a TTR-directed small interfering RNA, and a TTR-directed antisense oligonucleotide, along with supportive therapies. Gene-silencing Therapies. hATTR market : In The Pipeline.
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