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RNAi

This page shows the latest RNAi news and features for those working in and with pharma, biotech and healthcare.

ACPs to lead gene therapies in haemophilia market growth

The two drugs have distinct mechanisms – Hemlibra is a bispecific factor IXa- and factor X-directed antibody, while fitusiran is an RNA interference (RNAi) therapeutic targeting the endogenous anticoagulant antithrombin (AT) –

Latest news

Alnylam gains key NICE approval for Onpattro

The thumbs up from England’s cost effectiveness watchdog is an important milestone for Alnylam and Onpattro, its first marketed drug and the first-of-its-kind RNAi gene silencing treatment. ... Onpattro is expected to have the upper hand on its rival
Twentyeight-Seven raises another $15m for non-coding RNA drugs

This is a different approach to the emerging class of RNA therapeutics that target RNA that is directly involved in protein expression, such as antisense and RNA interference (RNAi) drugs from
Biotech now vital to pipelines – but success rates falling

first RNA interference (RNAi) therapy and Lox/Bayer’s NTRK gene fusion – targetting Vitrakvi (larotrectinib).
Alnylam swaps Sanofi for Regeneron as R&D partner

That includes pairing Regeneron's phase 1 antibody pozelimab (REGN3918) with Alnylam’s phase 2 RNAi drug cemdisiran, both of which target the complement C5 pathway, including other antibody/RNAi combinations. ... parties. It also reinforces the
Sanofi exits R&D pact with Alnylam on rare disease

Genzyme had previously licensed rights in 2012 to Alnylam’s lead RNAI drug Onpattro (patisiran) for hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), which became the first drug in the RNAi class ... The R&D deal was tweaked in January

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Latest Intelligence

Deal Watch October 2018

Dicerna/. Lilly. GalXC RNAi technology platform for targets in Cardiometabolic disease, neurodegeneration and pain.
Alnylam’s big moment: bringing groundbreaking RNAi drugs to Europe

This has been Alnylam’s story over the past 16 years, as it built on Andrew Fire and Craig Mello’s Nobel prize winning discovery that naturally occurring RNAi “silences” genes, ... Its RNAi technology can be used to target any gene expressed in
Boosting pharma’s metabolic rate

New frontiers such as cell therapy and RNAi are becoming therapeutic realities and exciting new challenges are coming over the horizon, creating a constantly shifting landscape that leaders must navigate.
Deal Watch September 2016

800. Arrowhead / Amgen. Licences. 2 agreements to develop and commercialise RNAi therapies in CVS disease, upfront payments of $35m, $21.5m in equity plus $617m in option payments. ... 95. Geron / J&J. Licence. Exclusive worldwide rights to Geron's
Pharma deals during January 2014

The deal comprised a range of RNAi assets including pre-clinical candidates, IP and delivery technologies. ... The other deal announced by Alnylam was the expansion of its deal with Sanofi (Genzyme unit_ to develop RNAi therapeutics in rare diseases.

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Latest appointments

Bruce Peacock joins Dicerna from Ophthotech

Massachusetts-headquartered Dicerna develops RNAi-based therapeutics with a focus on the discovery and development of treatments for rare inherited diseases involving the liver and for cancers that are genetically defined.
Silence appoints chief medical officer

These studies involve Silence's investigational portfolio of RNA interference (RNAi) therapeutics, which aim to treat conditions by selectively turning off genes expressed in an individual with a certain disease.

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Latest from PMHub

How do you define a biotech company?

This approach is particularly apparent in some of the recent examples of technology pioneered by biotech organisations, such as CAR-T technology (Novartis, Juno and Kite Pharma) and RNAi (Alnylam) where

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