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Yair Einhorn Profile picture
Yair Einhorn
@yaireinhorn
#Science & #Innovation. Special interest in #BioTech, #GeneEditing, #EpiGeneticEditing, #GeneTherapy 🧬 #CRISPR & #Ophthalmology. #Markets & #Stocks 📈
Mike Hargett Profile picture 1 subscribed
Sep 17 • 11 tweets • 5 min read
1/@BeamTx has recently reported new preclinical data demonstrating the ability of its in vivo drug candidate $BEAM-302 to directly correct the PiZ mutation - the primary disease-causing mutation associated with severe alpha-1 antitrypsin deficiency (AATD). #BioTech #CRISPR $XBI Beam Therapeutics - a biotechnology company developing precision genetic medicines through base editing, today reported new preclinical data demonstrating the ability of its in vivo drug candidate, BEAM-302, to directly correct the PiZ mutation, the primary disease-causing mutation associated with severe alpha-1 antitrypsin deficiency (AATD). 2/Alpha-1 antitrypsin deficiency (AATD) is the lack of a protein made by the patient’s liver. AATD is a genetic disease that can affect both the patient’s liver or the lung. Lung problems typically begin at the age of 20-50 and can include shortness of breath, wheezing, or an increased risk of lung infections.
Alpha-1 antitrypsin deficiency (AATD) is the lack of a protein made by the patient’s liver. AATD is a genetic disease that can affect both the patient’s liver or the lung. Lung problems typically begin at the age of 20-50 and can include shortness of breath, wheezing, or an increased risk of lung infections.
Feb 25 • 17 tweets • 25 min read
1/As promised & after reading $CRSP latest Q4 2022 #financial #report here is my impression regarding @CRISPRTX latest corporate status. I have focused only on the main issues that I found to be the most interesting & relevant. #CRISPR #BioTech #FinTwit #Genomics #GeneEditing 🧵 CRISPR Therapeutics, a biopharmaceutical company focused on 2/IMO the most significant corporate event in Q4 was $CRSP announcement that regulatory Exa-cel submissions of both #SickleCell & #BetaThalassemia validated in the #EU & #UK & that the @US_FDA BLA submission is on track by the end of Q1 ‘23 - possibly reaching the #markets in ‘23 Regulatory submissions complete for exagamglogene autotemcel
Feb 23 • 5 tweets • 4 min read
1/@GraphiteBio announced that after reviewing its business, it decided to discontinue any further development of nulabeglogene autogedtemcel (nula-cel) for #SickleCell disease, to explore a range of strategic alternatives & to reduce 50% in its workforce! #CRISPR #BioTech $GRPH Graphite Bio today announce... 2/@GraphiteBio is also exploring the potential to continue nula-cel development externally. $GRPH intends to continue research activities associated with its early-stage non- genotoxic conditioning program, with the goal of advancing toward potential development candidates.👇
Feb 22 • 5 tweets • 6 min read
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...
Dec 13, 2022 • 7 tweets • 11 min read
1/@GraphiteBio presented preclinical results supporting the use of a single-#cell #RNA #sequencing method to assess #gene correction outcomes in #patients treated with nulabeglogene autogedtemcel (nula-cel) - GPH-101. #BioTech #CRISPR #GeneEditing #Genomics $GRPH #ASH22 GraphiteBio presented precl... 2/GPH101 (nula-cel) is a #CRISPR #Cas9 #GeneEditing autologous #stem #cell-based #therapy in clinical development aimed to treat #SickleCellDisease. GPH101 is designed to directly correct the underlying mutation, thereby decreasing HbS production & restoring HbA expression #ASH22 GPH101 (nula-cel) is a CRIS...
May 21, 2022 • 14 tweets • 14 min read
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