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Biotech / Medical : Regeneron Pharmaceuticals -- Ignore unavailable to you. Want to Upgrade?

To: Miljenko Zuanic who wrote (3335)	7/16/2021 3:46:26 PM
From: Felix B	Read Replies (1) \| Respond to of 3557

You sound upbeat with your ANLY and REGN collaboration so why do you not predict a lot of candidates from them?

To: Miljenko Zuanic who wrote (3335)	7/19/2021 3:29:47 PM
From: CuttingEdge Bio	Read Replies (1) \| Respond to of 3557

What a strange comment. Alnylam's platform is designed for gene silencing, and their proven products (onpattro, givlaari, oxlumo) target gene silencing not gene correction. They are downregulating protein expression by silencing the gene expression through degrading mRNA. This is what REGN signed up for. It would be particularly useful for intracellular targets you want to shut off that may not be reached by antibodies. (Hence HSD). So you can shut off protein production even though you can't bind the proteins with antibodies. But there is another angle to this and it doesn't necessarily require a combo for differentiation from an antibody approach. GPR75 is indeed a G-coupled protein receptor, however it is located primarily in the hypothalamus. Plain vanilla antibodies are not very good to use if your target is in the brain. Despite many companies pursuing amyloid antibodies over and over again, the fact remains that for most of them almost none of the antibody in circulation actually gets to the brain. Roche has worked on a blood brain barrier shuttle technology for its latest amyloid approach that might shuttle it better. Alnylam showed some proof of concept for targeted delivery to the eye and CNS, and this is what prompted the REGN deal with them in 2019 for a broad collaboration. This large deal happened later after they had already previously collaborated with ALNY for HSD in NASH and potentially other targets in liver. The point is that if an antibody won't do the job in the brain, then RNAi might be the preferable choice when it is targeted to CNS. Maybe you don't believe Alnylam's data. That's ok. REGN believed it enough to sign onto this major collaboration.