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Yair Einhorn Profile picture
@yaireinhorn
May 21, 2022 • 14 tweets • 14 min read • Read on X
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1/As promised & after reading $NTLA latest Q1 2022 financial report here is my analysis regarding @intelliatx latest corporate status. I have focused only on the main issues that I found to be the most interesting & relevant. #CRISPR #GeneEditing #BioTech #FinTwit #Genomics👇
2/NTLA-2001 is the most advanced In-Vivo #CRISPR/#Cas9 #GeneEditing program in $NTLA portfolio & is aimed to treat Transthyretin Amyloidosis - (ATTR). NTLA-2001 could potentially halt & reverse the disease in a “one time” treatment by knocking out the TTR gene with a single dose
3/In February $NTLA has presented an updated clinical data from its Phase 1 study of NTLA-2001 which included 15 patients with ATTR amyloidosis. IMO NTLA-2001 readout looks promising & here is a summary that I wrote shortly after @intelliatx’s presentation of NTLA-2001👇
4/NTLA-2002 is aimed to knock out the KLKB1 gene in the #liver thus reducing total plasma kallikrein protein & activity, a key mediator of #HAE - hereditary #angioedema. In 12/1/21 $NTLA has dosed the 1ST patient & will publish interim data from the Phase 1/2 study in 2H of 2022.
5/Two more In-Vivo programs are $NTLA 3001 & 2003: NTLA-3001 is aimed for AATD-associated #Lung disease & IND will be filled during 2023. NTLA-2003 is aimed for AATD-associated #Liver disease & @intelliatx is advancing towards IND-enabling activities for this program as well.
6/NTLA-5001 is an autologous T-cell therapy aimed for the treatment of all genetic subtypes of #AML. In March @intelliatx announced that the first patient was dosed in its Phase 1/2a study & that @US_FDA has granted orphan drug designation to NTLA-5001 for the treatment of #AML👇
7/IMO the most important development was the designation of NTLA-6001 - an allogeneic CAR-T program for treating hematologic #cancer including Hodgkin #lymphoma (cHL). NTLA-6001 is the first program that was developed using @intelliatx’s proprietary cell engineering platform👇
8/One of @intelliatx’s major strengths is its multiple collaborations: 1)acquisition of Rewrite 2)@OnkTherapeutics - the co - development of #CRISPR-edited NK #CellTherapy for #Cancer. 3)@KyvernaT - the development of KYV-201 - a CD19 CAR-T cell for an #autoimmune diseases.👇
9/As of 3/31/22 $NTLA had capital resources of $994.7M & R&D expenses of $133.1M. IMO although the cash burn has increased by $94M, @intelliatx cash position of $994M will enable it to both continue to develop its pipeline & to initiate additional acquisitions & collaborations.
10/Upcoming Milestones:
1.NTLA-2001 - ATTR:
ATTRv-PN arm - Phase 1 data in June 2022
ATTR-CM arm - Phase 1 data in 2H 2022
2.NTLA-2002 - HAE:
Phase 1/2 data in 2H 2022 3.NTLA-3001 - AATD:
File an IND in 2023
4.NTLA-5001 - AML:
Enrolment of patients in Phase 1/2a during 2022
11/@intelliatx is definitely an impressive & promising #GeneEditing company. Both $NTLA proprietary pipeline & its systematic strategy of collaborations & acquisitions are the key factors which defines $NTLA as a leading #CRISPR and #GeneEditing company.
on a personal note: one of my main interests in $NTLA was its collaboration with @SparingVision - aimed to develop #Ocular #GeneEditing platforms for the treatment of #IRDs. As Of Q1 22 no progress was published yet & I eagerly await to see what progress this agreement will bring
And as always please feel free to share and retweet so that those on @Twitter or #FinTwit who are interested in #GeneEditing, #CRISPR, #BioTech & #Genomics will get this relevant data. Enjoy the rest of your #Weekend!
@ArielDa58728507

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More from @yaireinhorn

Yair Einhorn Profile picture
Aug 18, 2024
1/This great @WSJ’s 📊 shows how after spending tens of billions of dollars for a single BioTech company, big Pharma moguls such as $ABBV, $AZN, $BMY, $AMGN, $PFE & $VRTX have all shifted to acquiring smaller targets of >$5B - for both regulatory & financials reasons. $XBI 🧵👇 After shelling out tens of billions of dollars for single biotech companies, pharma- ceutical giants such as AbbVie, AstraZeneca and Merck have shifted to smaller targets cost- ing $5 billion or less. Many of the companies getting taken out are private. The reason, according to ex- ecutives, bankers and lawyers: Smaller deals are just easier to do in the current regulatory environment, and the sector is looking pretty picked over.
2/All 17 deals made by big Pharma during the first 6 months of ‘24 were $5B or less. During the same period last year, big Pharma made 9 deals, including 2 that were $10B or more. 9 of the 17 deals were for privately held companies, compared to 1 during the same period in 2023. All 17 deals announced by big pharmaceutical companies during the first six months of the year were valued at 5 billion dollars or less, according to re- search firm DealForma. During the same period last year, big drugmakers agreed to nine deals, including two that were 10 billion dollars or larger. Nine of the 17 deals were for privately held companies, compared with one during the same period last year, DealForma said.
3/Last year’s big acquisitions were led by @pfizer’s $43B purchase of cancer biotech $SGEN ❤️👇. By contrast & to demonstrate how things have changed, the biggest deal so far in 2024 was @VertexPharma $4.9B purchase of Alpine Immune Sciences & its experimental kidney drug. $PFE
Read 14 tweets
Yair Einhorn Profile picture
Jul 22, 2024
The biggest problem of Gene Editing & Gene therapy is delivery - how to deliver a genetic payload to the exact desired location. Here’s a great Infograph which shows which BioTech companies use Viral delivery methods (AAVs, LVs) compared to non-viral platforms (NLPs). $XBI The biggest problem of Gene Editing & Gene therapy is delivery - how to deliver the genetic payload to the exact desired location. Here is a great Infograph which shows which BioTech companies use Viral (AAVs) delivery methods compared to non-viral platforms (NLPs).
2/Must of the companies - as you can see in the graph m☝️- use a viral based delivery platform for Gene Therapy / Gene Editing treatments, mostly AAV - Adeno-Associated Virus based delivery. AAV is a naturally occurring virus being transformed into a delivery mechanism by replacing its viral DNA with new DNA, thus making it a precisely coded vector & it is no longer considered a virus, as most of its viral components have been replaced.
3/The main problem with AAV delivery system is that it is limited to the size of the payload which can fit into the virus vassal. It could also cause the patient an inflammatory response due to this external virus activity. This led to a search for a non-viral mechanism. $XBI The main problem with AAV delivery system is that it is limited to the size of the payload which can fit into the virus vassal. It could also cause the patient an inflammatory response due to this external virus activity. This led to a search for a non-viral mechanism. $XBI
Read 8 tweets
Yair Einhorn Profile picture
Jul 9, 2024
1/@nvelop_tx has presented its proprietary delivery system - DLVR-M, which has successfully demonstrated improved delivery of CRISPR, base editing & prime editing platforms to different human cell types. DLVR-M could be the key in overcoming the obstacle of CRISPR delivery. $XBI nvelop therapeutics has presented its proprietary delivery system - DLVR-M, which has successfully demonstrated improved delivery of CRISPR, base editing & prime editing platforms to different human cell types. DLVR-M could be the key in overcoming the obstacle of CRISPR delivery
2/Every Gene Therapy is combined from 2 components - the genetic payload which is the mechanism for fixing the genetic disease or the cure itself & a delivery vehicle which needs to deliver the “package” to a specific human tissue & location. Just Imagine a pickup truck & a box. Image
3/As of today the only approved delivery platform for Gene Therapy / Gene Editing is AAV - Adeno-Associated Virus based delivery. AAV is a naturally occurring virus being transformed into a delivery mechanism by replacing its viral DNA with new DNA thus making it a precisely coded vector & it is no longer considered a virus, as most of its viral components have been replaced
Read 12 tweets
Yair Einhorn Profile picture
May 29, 2024
1/WOW! hC Bioscience announces its first ever tRNA-based program for Duchenne muscular dystrophy (DMD). hC is developing anticodon engineered tRNAs as a potential treatment for DMD patients with shortened & nonfunctional dystrophin due to premature termination codons (PTCs). $XBI WOW! hC Bioscience announces its first ever tRNA-based program for Duchenne muscular dystrophy (DMD). hC is developing anticodon engineered tRNAs as a potential treatment for DMD patients with shortened & nonfunctional dystrophin due to premature termination codons (PTCs).
2/Duchenne is a severe progressive disease which rapidly worsening children’s muscle function often using a wheelchair by early adolescence & eventually needing artificial ventilation to breathe. DMD is caused by mutations to dystrophin that affect about 300,000 males worldwide & PTCs account for approximately 26% of cases.Duchenne is a severe progressive disease which rapidly worsening children’s muscle function often using a wheelchair by early adolescence & eventually needing artificial ventilation to breathe. DMD is caused by mutations to dystrophin that affect about 300,000 males worldwide & PTCs account for approximately 26% of cases.
3/A nonsense mutation or premature termination codons (PTCs) is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in DuchenneA nonsense mutation or premature termination codons (PTCs) is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne
Read 7 tweets
Yair Einhorn Profile picture
May 21, 2024
1/As promised and after reading $CRBU latest Q1 2024 financial report here is my impression regarding @CaribouBio latest corporate status. As always - I have focused only on the main issues that I found to be the most interesting & relevant. Here’s my summary 🧵👇 $XBI #BioTech As promised and after reading Caribou’s latest Q1 2024 financial report here is my impression regarding Caribou Bio latest corporate status. As always - I have focused only on the main issues that I found to be the most interesting & relevant. Here’s my summary
2/IMO the most significant corporate event was $CRBU decision - due to internal portfolio prioritisation - to terminate the development of its CB-020 program - a preclinical allogeneic anti-POR1 CAR-NK cell therapy - a decision which narrowed dramatically the company’s pipeline. In my opinion the most significant corporate event was Caribou’s decision - due to internal portfolio prioritisation - to terminate the development of its CB-020 program - a preclinical allogeneic anti-POR1 CAR-NK cell therapy - a decision which narrowed dramatically the company’s pipeline.
3/Another major event was $CRBU announcement that it had received FDA clearance of its Investigational New Drug - IND application to evaluate CB-010 ability to treat patients with lupus nephritis (LN) & extrarenal lupus (ERL) Thus expending CB-010’s therapeutic potential. $XBI
Another major event was Caribou’s announcement that it had received FDA clearance of its Investigational New Drug - IND application to evaluate CB-010 ability to treat patients with lupus nephritis (LN) & extrarenal lupus (ERL) Thus expending CB-010’s therapeutic potential
Read 18 tweets
Yair Einhorn Profile picture
May 8, 2024
1/@LineageCell presented promising data at the @FightBlindness Gene Therapy innovation summit - as part of #ARVO2024, from its clinical study of RG6501 (OpRegen) - a retinal pigment epithelial Cell therapy aimed to treat age-related macular degeneration dry AMD. $LCTX $RHHBY $XBI Lineage cell has presented promising data at the foundation fighting blindness Gene Therapy innovation summit - as part of ARVO2024, from its clinical study of RG6501 (OpRegen) - a retinal pigment epithelial Cell therapy aimed to treat age-related macular degeneration dry AMD
2/@LineageCell has developed a unique technology that enables it to transplant specific cell types from a single pluripotent cell line thus creating “off the shelf” cell transplants platform for multiple conditions. $LCTX most advanced program is its OpRegen ocular platform. $XBI Lineage cell has developed a unique technology that enables it to transplant specific cell types from a single pluripotent cell line thus creating “off the shelf” cell transplants platform for multiple conditions. Lineage’s most advanced program is its OpRegen ocular platform.
3/Dry AMD leads to the loss of retina cells thus creating an area of geographic atrophy-GA, which leads to impaired vision & blindness. By using a subretinal injection of RPE cells into the eye OpRegen will be able to preserve or improve a patient’s vision
Read 12 tweets

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