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Yair Einhorn Profile picture
@yaireinhorn
Feb 22, 2023 • 5 tweets • 6 min read • Read on X
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1/@LineageCell announced it entered an exclusive option & license agreement with $ERNA for the development of novel B2M deficient induced pluripotent #stem #cell #iPSC by using Eterna’s #GeneEditing technology thus developing therapies for #CNS diseases. #BioTech #CRISPR $LCTX Lineage Cell Therapeutics, ...
2/The new cell lines - to be developed by $ERNA, will support the creation of additional product candidates at $LCTX and specifically for the treatment of central #nervoussystem #CNS disorders & other #neurology targets via Eterna’s IP #CRISPR collaboration with @FactorBio The new cell lines to be de...
3/This agreement will also allow @LineageCell to enter the #CRISPR field and will provide $LCTX an option to obtain an exclusive license to utilize & sublicense $ERNA novel #GeneEditing cell lines for #preclinical, #clinical, & commercial purposes in the field of CNS diseases. This agreement will also al...
4/A feature of the starting #cell line is the targeted deletion of the B2M gene, which is designed to reduce the immunogenicity of product candidates derived from the lines by inhibiting rejection by CD8+ T cells. 4/A feature of the starting...
5/@LineageCell & $ERNA intend to initiate additional planned #GeneEditing platforms that may further differentiate the cell line from others currently in use by competitors. Financial terms of the collaboration between $LCTX & $ERNA were not disclosed.

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More from @yaireinhorn

Yair Einhorn Profile picture
May 8
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
Yair Einhorn Profile picture
Apr 30
1/@WaveLifeSci today announced the approval of its first clinical trial application (CTA) for its RestorAATion-2 clinical trial of $WVE-006, Wave’s first-in-class RNA editing oligonucleotide, which is being developed for the treatment of alpha-1 antitrypsin deficiency-AATD. $XBI Wave Life Sciences - a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced the approval of its first clinical trial application (CTA) for its RestorAATion-2 clinical trial of WVE-006, the company’s first-in-class RNA editing oligonucleotide, which is being developed for the treatment of alpha-1 antitrypsin deficiency (AATD).
2/WVE-006 is a first-in-class, GalNAc-conjugated RNA editing oligonucleotide aimed to correct the single base mutation in messenger RNA (mRNA) coded by the SERPINA1 Z allele, thereby enabling restoration and circulation of functional, wild-type alpha-1 antitrypsin (M-AAT) protein WVE-006 is a first-in-class, GalNAc-conjugated RNA editing oligonucleotide aimed to correct the single base mutation in messenger RNA (mRNA) coded by the SERPINA1 Z allele, thereby enabling restoration and circulation of functional, wild-type alpha-1 antitrypsin (M-AAT) protein
3/RestorAATion-2 is a Phase 1b/2a open label study designed to evaluate the safety & tolerability of WVE-006 in individuals with AATD who have the homozygous Pi*ZZ mutation. $WVE remains on track to deliver proof-of-mechanism data - restoration of M-AAT protein in serum, in 2024. RestorAATion-2 is a Phase 1b/2a open label study designed to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of WVE-006 in individuals with AATD who have the homozygous Pi*ZZ mutation. The trial includes both single ascending dose (SAD) and multiple ascending dose (MAD) portions. The company remains on track to deliver proof-of-mechanism data, as measured by restoration of M-AAT protein in serum, in 2024.
Read 6 tweets
Yair Einhorn Profile picture
Apr 2
1/@VerveTx announced that due to observed laboratory abnormalities associated with $VERV-101, it has decided to pause enrollment in its Heart-1 clinical trial. Verve is conducting an investigation & will work with regulatory authorities to define a path forward for VERVE-101 $XBI Verve therapeutics announced today that due to observed laboratory abnormalities associated with VERV-101, it has decided to pause enrollment in its Heart-1 clinical trial. Verve is conducting an investigation & will work with regulatory authorities to define a path forward for VERVE-101
2/VERVE-101 is being evaluated in the Heart-1 Phase 1b clinical trial with trial endpoints of safety and tolerability as well as changes in blood PCSK9 protein and low-density lipoprotein cholesterol (LDL-C) levels in patients living with heterozygous familial hypercholesterolemia (HeFH), established atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemiaVERVE-101 is being evaluated in the Heart-1 Phase 1b clinical trial with trial endpoints of safety and tolerability as well as changes in blood PCSK9 protein and low-density lipoprotein cholesterol (LDL-C) levels in patients living with heterozygous familial hypercholesterolemia (HeFH), established atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia
3/6 participants have been dosed at 0.45 mg/kg of VERVE-101 from a total of 13 participants. For the first 5 in the 0.45 mg/kg cohort (with follow-up of >28 days) $VERV-101 demonstrated time-averaged LDL-C reductions ranging from 21% to 73%, & averaging 46% (cut-off date 3/18/24)
Read 7 tweets
Yair Einhorn Profile picture
Mar 28
1/@CaribouBio announced that preclinical data from its CB-012 program - an allogeneic anti-CLL-1 CAR-T cell therapy aimed to treat relapsed or refractory acute myeloid leukemia (r/r AML), will be presented at the upcoming #AACR24 held April 5-10, 2024 in San Diego. $CRBU #CRISPR Caribou Biosciences- a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today announced that preclinical data from CB-012, an allogeneic anti-CLL-1 CAR-T cell therapy for the treatment of relapsed or refractory acute myeloid leukemia (r/r AML), will be presented at the upcoming American Association for Cancer Research (AACR) Annual Meeting 2024, held April 5-10, 2024 in San Diego.
2/CB-012 is an anti-CLL-1 CAR-T cell therapy engineered with five genome edits, enabled by @CaribouBio’s patented next-generation CRISPR technology platform, which uses Cas12a chRDNA Gene Editing platform to significantly improve the specificity of genome edits. $CRBU CB-012 is an anti-CLL-1 CAR-T cell therapy engineered with five genome edits, enabled by Caribou’s patented next-generation CRISPR technology platform, which uses Cas12a chRDNA Gene Editing platform to significantly improve the specificity of genome edits.
@CaribouBio 3/CB-012 is the first allogeneic CAR-T cell therapy with both checkpoint disruption, through a PD-1 knockout, and immune cloaking, through a B2M knockout and B2M–HLA-E fusion protein insertion; both armoring strategies are designed to improve antitumor activity. CB-012 is the first allogeneic CAR-T cell therapy with both checkpoint disruption, through a PD-1 knockout, and immune cloaking, through a B2M knockout and B2M–HLA-E fusion protein insertion; both armoring strategies are designed to improve antitumor activity.
Read 5 tweets
Yair Einhorn Profile picture
Mar 11
1/As promised and after reading $VERV latest Q4 & full year 2023 financial report here is my impression regarding @VerveTx 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🧵👇 $XBI #BioTech Verve Therapeutics - a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today reported pipeline updates and financial results for the fourth quarter and year ended December 31, 2023.
2/IMO the most significant corporate event was @VerveTx announcement that the @US_FDA has cleared its Investigational New Drug (IND) Application for $VERV-101 in patients with HeFH. Verve is currently working to activate U.S. trial sites & intends to dose the first patient with VERVE-101 in the US along with its ongoing UK & New Zealand clinical sites.In my opinion the most significant corporate event was Verve’s announcement that the FDA has cleared its Investigational New Drug (IND) Application for VERV-101 in patients with HeFH. Verve is currently working to activate U.S. trial sites and intends to dose the first patient with VERVE-101 in the US along with its ongoing UK and New Zealand clinical sites.
@VerveTx @US_FDA 3/@VerveTx’s leading clinical program remains VERVE-101. $VERV-101 is a Gene Editing platform which uses Base editing to treat heterozygous hypercholesterolemia (HeFH) patients by permanently turning off the PCSK9 gene in the Liver and thus reducing disease-driving LDL-C. Verve’s leading clinical program remains VERVE-101. VERV-101 is a Gene Editing platform which uses Base editing to treat heterozygous hypercholesterolemia (HeFH) patients by permanently turning off the PCSK9 gene in the Liver and thus reducing disease-driving LDL-C.
Read 17 tweets
Yair Einhorn Profile picture
Mar 4
1/As promised & after reading $NTLA latest Q4 & full year 2023 financial report here is my impression regarding @intelliatx latest corporate status. As always I have focused only on the main issues that I found to be the most interesting & relevant. Here is my 🧵👇 $XBI #BioTech Intellia Therapeutics - a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today reported operational highlights and financial results for the fourth quarter and year ended December 31, 2023.
2/IMO the most significant corporate event was @intelliatx collaboration signed with @ReCodeTx - which uses tissue-specific delivery to power mRNA & gene correction therapeutics to develop novel medicines for the treatment of Cystic fibrosis based on $NTLA Gene Editing platform In my opinion the most significant corporate event was intellia’s collaboration signed with ReCode therapeutics - which uses tissue-specific delivery to power mRNA and gene correction therapeutics to develop novel medicines for the treatment of Cystic fibrosis based on intellia’s platform
3/Another major development - which was recently presented in the @jpmorgan’s health conference, was @intelliatx’s restructured pipeline & its new 2024-2026 main Strategic Priorities:
1. Trials for its 2 in vivo #CRISPR platforms - $NTLA-2001 & 2002
2. POC for $NTLA new CRISPR-in vivo targeted gene insertion & allogeneic ex vivo program
3. Developing new Gene editing programs outside the liver
4. DNA writing3/Another major development - which was recently presented in the jpmorgan’s health conference, was intelliatx’s restructured pipeline and its new 2024-2026 main Strategic Priorities: 1. Trials for its 2 in vivo CRISPR platforms - NTLA-2001 & 2002 2. POC for NTLA new CRISPR-in vivo targeted gene insertion and allogeneic ex vivo program 3. Developing new Gene editing programs outside the liver 4. DNA writing
Read 18 tweets

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