RARECast: Sangamo Advances Gene Therapies for Multiple Rare Diseases Into Clinical Development
The promise of gene therapy has rare disease patients not only contemplating the potential of new treatments, but ones that can free them from chronic therapies and potentially provide cures. Sangamo Therapeutics, long pursuing its proprietary gene editing technology, is suddenly moving into the clinic with four experimental therapies including treatments for hemophilia A, hemophilia B, MPS I, and MPS II. The company is currently enrolling three trials and expects to begin enrollment on a fourth trial later this month. We spoke to Sandy Macrae, President and CEO of Sangamo, about the diseases it is targeting, the company’s unique approach to gene therapy, and it’s strategy for moving their therapies through clinical development and to the market.
Q2 earnings after hours today. the numbers are much less important than the commentary re trials IMO.
The upfront from PFE should be recognized this quarter so the earnings will look good relative to the street. Similar to PFE expensing it, SGMO should record it as income unless there were material contingencies or deliverables associated with the upfront (which have not been disclosed).
PFE CC: I want to point out that our updated adjusted diluted EPS guidance range absorbs $75 million of adjusted research and development expenses that were recorded in the second quarter 2017 due to our agreement with Sangamo Therapeutics, which we announced in May 2017 to develop and commercialize gene therapy programs for hemophilia A.
As we noted at – advance in the field, we recently licensed, in a partnership with Sangamo, a factor VIII gene therapy, which is quite a big market. We're just embarking on clinical studies with that. And we are exploring to take a step into really difficult diseases like Duchenne's muscular dystrophy. We're planning our internal program to go into the clinical study early next year. So as Ian alluded to, we decided to move from more incremental to more transformative therapies, and so far are very encouraged about the quality of these technologies. ===================== Assuming around $20-25m in opex vs the $70m upfront the profit this quarter should be a nice headline.
Amazing science..this right here is why engineered proteins are much better than fast proteins like CRISPR...they can be modified and engineered the way you want them..
For example..why do you need to skip some base pairs? because not every human has the same exact sequence even though their phenotype is the same..this way you can design a ZFN that skips the variable base pairs but still targets the gene in an exact manner...CRISPR would have to create a new mRNA for each human to get the same result.. Also skipping base pairs allows for choosing the ZFN that have tighter binding than other ones
Why reverse the ZFP order so you can target both sides of the chain? because ZFN have greater affinity for some base pairs than other ones, and if all those base pairs are on one side of the chain, you can engineer it so it binds on the same side instead of opposite..this lead to more specific and tight binding and thus probably better cutting and efficiency as well
Last Edit: Apr 4, 2018 9:09:29 GMT -6 by spaddyman
I'm really curious how these presentations are going to be used. The clarity of approach and accelerating improvement in their platform is stunning but this is too much for a normal presentation format. Wonder if they have an analyst day planned? Maybe for their bus dev team given the volume of outside interest?
No wonder PFE decided to transfer their CLLS targets to Allogeneic. They have first hand insight so why continue invest resources in a plodding competitor when SGMO this kind of progress
Yah they dont have specific dates on the presentations so guessing its just data sheets based on your specific interest..Every time i read the same data i seem to understand it more as i learn more about gene editing and gene therapy in general haha
Yes PFE bailed..if it was awesome they wouldn't have given it up just for a 25% stake in new company..They probably saved themselves a Billion dollars trying to get these through trials.. I think the KITE guys know SGMO better but couldn't turn down the idea of starting another hot CART company where investors are willing to put in hundreds of millions of dollars
Recap of Kite upfront, HIV grant, equity raise and rationale of investing in opportunities as previously described.
18 month milestones include data from 7 trials, primate data, build-out of HQ/mfg and pipeline expansion
Finance largely a repeat but did say the current cash is expected to last 5 yrs
SB-525 6 potential cohorts/Up to 20 patients/4 treated to date/data late summer.
SB-318 4 patients screened/First treatment soon/up to 9 patients in 2 dose cohorts (based upon MPS 1)/data C18
SB-Fix 4 sites active/continue to screen for eligible patients/up to 12 patients in 3 cohorts/UK study to initiate YE18
SB-400 first site open/6 patients in 1 cohort/first subject enrollment expected 1H18
Fabry IND expected "this year"
Preview of 2018 ASGCT IMD • Liver-Based Expression of the Human Alpha-Galactosidase A Gene in a Murine Fabry Model Results in Continuous Therapeutic Levels of Enzyme Activity and Effective Substrate Reduction
• ZFN-Mediated In Vivo Genome Editing Results in Therapeutic Levels of α-Galactosidase A and Effective Substrate Reduction in Fabry Knockout Mice
Immunology • Highly Efficient and Specific Multiplexed Gene Editing in T Cells using Enhanced Zinc-Finger Nucleases (ZFNs) Enables Strategic Engineering of Allogeneic T Cell Immunotherapies
CNS • Designed Zinc Finger Protein Transcription Factors for Single-Gene Regulation Throughout the Central Nervous System
Technology and delivery • Enhancing ZFN Expression Construct and Nuclease Activity Leads to Improvements of In Vivo Genome Editing Platform
• Global and Tunable Suppression of Zinc Finger Nuclease and ZFP-Transcription Factor Off-Target Activity via Discrete Framework Substitutions
• Non-Viral Delivery of ZFN mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets
Q: Is treatment of addl patients similar to the first in HemoA? Avoided any info awkwardly. Said they continue with program which you can use to draw your own conclusions.
Kite/Gilead collaboration Q: Still analyzing. Will be straight-line but need to project performance period. Update next quarter.
How will you present data? We're looking at some medical conferences.
Efficacy question was this a delay re summer vs midyear: A: no change just waiting for data to mature as stated before.
Liver biopsy data: Looking at enzyme levels now to avoid biopsy until later in program.
Statement on in vivo editing: effectiveness of ZFs is not in question, testing the AAV delivery.
Huntington's silencing and Q on Tekada acquisition and potential for return of program? should only be good news/Hear from Shire that the program continues.
Under: Been on the sidelines for a bit holding (building) cash. Now that "BIGLEY" has rolled out the tax plan its time to jump in.
Dec 21, 2017 19:06:02 GMT -6
martyc: Looks like you are buying Msft again!
Dec 15, 2017 11:23:29 GMT -6
martyc: The news that Trump called Rupert to congratulate him sure seems to indicate that this is heading to approval
Dec 15, 2017 11:22:23 GMT -6
Under: DIS finally getting some traction.?
Dec 14, 2017 17:08:45 GMT -6
martyc: I took an entry level position in DIS. Will add eventually to overweight when it becomes clearer that the deal will go thru. Can't believe how well positioned they will be. 60% Hulu. 20% of content watched on NFLX they can pull. More in thread
Dec 14, 2017 11:05:16 GMT -6
Under: Great posts on $DIS
Dec 13, 2017 17:50:49 GMT -6
Under: $ROKU Citron on a war path.
Nov 28, 2017 15:11:20 GMT -6
Under: $HAS takeover bid for $MAT?
Nov 10, 2017 16:16:07 GMT -6
martyc: Not looking like the market will provide any discounted opp for SGMO. Call was just too professional and all signs indicate they are on a great path for commercialization. Happy with core but wish I had some trading shs
Nov 10, 2017 9:04:05 GMT -6
martyc: For anyone looking to find an entry point into SGMO, I'm almost hoping is sells off in next few days so I can add more. They are really clicking but the fact they haven't signed new deals might cause some to exit. Watching as I have room for trading shs
Nov 9, 2017 18:28:09 GMT -6
martyc: Been an interesting ride so far. I figured the Bears would be about this good but hoped the O wouldn't look so lame. Another building yr but still possible to get to 8-8 IMO
Nov 9, 2017 18:26:08 GMT -6
Under: whats up with your Bears this year Marty?
Nov 9, 2017 17:35:25 GMT -6
martyc: Hope you were long ROKU. I wanted to see Q first so missed out
Nov 9, 2017 7:08:53 GMT -6