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Changed subject to add comments about BIIB collab targets and delivery. The prior post was the research article published in Mar 2021. Some context:
Comments from the 4Q19 earnings call transcript:
Biogen is committed to quickly progressing programs forward to IND applications
Preclinical studies have demonstrated the ability of AAV vectors to efficiently deliver ZFP-TFs that are targeted to tau or synuclein and results in highly specific, potent and tunable repression.
Preclinical data last year demonstrated significant reduction of tau expression in the NHP brain following admin of targeted ZFP-TFs.
Q: if BIIB does not select the nine unselected targets by 2025 what happens? A: If not selected by term of 5 yr agreement we would be freed from the exclusivity under the BIIB agreement. (me: suggests the 9 are known but unselected)
Q: How long does it take to get a therapy ready for a product/program? A: We probably need 9-18 months per target.
We have been working on synuclein and Prion for only 3-6 months and are pleased at how quickly and effectively the team has created assets for these new targets. One of the things that gave BIIB great confidence that we could succeed with their list. (me: again note "their list" which implies all targets are known)
Q: Will BIIB have exclusive rights to your novel AAV vectors? A: BIIB will have access to our novel capsid but on a non-exclusive basis, except when it comes to the target exclusively licensed to them. Very important consideration for the deal.
We have not disclosed updated timelines for BIIB targets. The next step for tau and synuclein is to conduct IND enabling studies but way too early to comment on timing for entering the clinic.
Q: Does your AAV stand out in that perspective (address all cell types in the brain, spinal cord and dorsal root ganglion) and neuro inflammation specifically as well as tropism? A: We look carefully for neuro inflammation both in CNS and peripheral nervous system. Particularly after that work that has come out over the past year in the dorsal root ganglion and that would be something that our vector did not cause for this to be a successful medicine and move forward. (me: likely the focus of ongoing safety efforts mentioned at several points)
Important to understand that the BIIB deal is non-exclusive. There is exclusivity on 12 targets including tau and synuclein. (Me: again a reference targets being known or they could not be exclusive.)
Q: Before selection of additional 9 targets, is there a certain level of preclinical data or in vitro data you will generate and a decision making time period? A: We cannot disclose a lot of details. We need to bring to BIIB a certain preclinical package or certain validation package and the deal is structured in a way that Sangamo is strongly incentivized to bring those packages as rapidly as possible to BIIB. (me: this statement is why I believe Target Selection Fees will be triggered sooner rather than later).