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Msg  34291 of 34731  at  5/2/2018 5:17:11 AM  by

egomaniakos


 In response to msg 34290 by  spaddy
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Re: Genevant website online

It is highly likely that developing their in house products is simply a proof of concept approach to entice collaborations and show that there is an alternative for the company if a potential collaborator does not want to collaborate. But Genevant would have technical know how issues as well as IP issues to commercialise such products with respect to the payloads.
 
As for SGMO getting an unlimited license, they may well do, but that is a financial consideration/choice. Usually companies start out with a limited number of licenses. A collaborator does not risk their technology being appropriated, no more than Genevant risks theirs being appropriated. These are normal business transactions.
 
However, you can be assured that Vivek will be ensuring that Genevant is well capitalised and extracting the best deal from SGMO or whomever else, especally if they want priority or exclusivity.
 
For example if SGMO doesn't want to deal then Genevant can turn to Bluebird that also used Acuitas to test the waters. I would suspect he would be quite happy to play companies off against each other. Below is the rather impressive ASGCT Bluebird LNP abstract:
 
 
In Vivo Gene Editing of the Murine Pcsk9 Locus Using Lipid Nanoparticle-Delivered Megatal mRNA 

Authors

John C. Moore1, Kyle Havens2, Seema Shah1, James Rottman1, Max Echterling2, Barbara Mui3, Madhumita Mahajan1, Aaron Edwards1, Ashley Gee1, Chih-Chi Chu1, Joe Barberio1, Jordan Jarjour2, Holly Horton1, Chris Barbosa3, Ying Tam3, Tom Redelmeier3, Jenny Marlowe1, Philip Gregory1, Mike Certo1

1Bluebird Bio, Inc., Cambridge, MA,2Bluebird Bio, Inc., Seattle, WA,3Acuitas Therapeutics Inc., Vancouver, BC, Canada

Body

Gene Editing of the Murine Pcsk9 Locus Using Lipid Nanoparticle-Delivered Megatal mRNAIn Vivo176 -
Author Block: John C. Moore1, Kyle Havens2, Seema Shah1, James Rottman1, Max Echterling2, Barbara Mui3, Madhumita Mahajan1, Aaron Edwards1, Ashley Gee1, Chih-Chi Chu1, Joe Barberio1, Jordan Jarjour2, Holly Horton1, Chris Barbosa3, Ying Tam3, Tom Redelmeier3, Jenny Marlowe1, Philip Gregory1, Mike Certo1 1Bluebird Bio, Inc., Cambridge, MA,2Bluebird Bio, Inc., Seattle, WA,3Acuitas Therapeutics Inc., Vancouver, BC, Canada
Disclosure Block: J.C. Moore: 1; Commercial Interest i.e. Company X; bluebird bio, Inc.. 1; For what role? i.e. Speaker; Employee.
MegaTALs are fusions between transcription activator-like (TAL) DNA binding domains and engineered meganucleases that can be reprogrammed to create double-strand breaks at a single genomic locus. Delivery of gene editing reagents to the liver via lipid nanoparticle (LNP)-encapsulated mRNA has emerged as a promising approach for in vivo genome editing, owing to the generation of robust yet transient protein expression in hepatocytes. To evaluate this approach, we reprogrammed a megaTAL to target the open reading frame of the murine proprotein convertase subtilisin/kexin type 9 (Pcsk9) gene. The Pcsk9 gene serves as an ideal in vivo gene editing proof of concept target because of its enriched liver expression and clinically validated role in cholesterol homeostasis, allowing for facile evaluation of editing by tracking PCSK9 protein and cholesterol serum levels. The Pcsk9 megaTAL demonstrates high in vitro editing activity, generating INDELs at greater than 80% of alleles when delivered to murine liver cell lines. To test the ability of the Pcsk9 megaTAL to edit hepatocytes in vivo, we formulated megaTAL mRNA into LNPs and intravenously administered the formulation to mice via tail vein injection. The formulations were well-tolerated, causing minimal elevation of liver transaminases at 24 hours with no indication of abnormal liver pathology. Dose-dependent editing was observed across 0.3 to 3.0 mg/kg ranges of mRNAs, with INDEL rates of up to 60% at the PCSK9 locus in bulk liver, resulting in an approximately 80% reduction of serum PCSK9 protein. Moreover, a significant dose-dependent reduction in total cholesterol of up to 50% was observed, concomitant to the reduction in PCSK9 serum protein and efficiency of gene disruption. These proof of concept data demonstrate the high rates of editing and therapeutic potential the megaTAL platform can achieve when delivered in vivo by LNP-encapsulated mRNA to hepatocytes.



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