#Science & #Innovation. Special interest in #BioTech, #GeneEditing, #EpiGeneticEditing, #GeneTherapy 🧬 #CRISPR & #Ophthalmology. #Markets & #Stocks 📈
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Apr 2 • 7 tweets • 3 min read
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
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 hypercholesterolemia
Mar 28 • 5 tweets • 2 min read
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
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
Mar 11 • 17 tweets • 7 min read
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
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.
Mar 4 • 18 tweets • 7 min read
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
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
Jan 30 • 6 tweets • 2 min read
1/@Tome_bio - a #GeneEditing company based on programmable genomic integration (PGI) platform has recently launched & raised $213M in Series A & B funding from leading investors: @a16zBioHealth, @GVteam, @FujifilmUS, #ARCHVenturePartners, @bruker & others. #BioTech #CRISPR $XBI
2/PGI - programmable genomic integration - combines the site-specificity of #CRISPR/Cas9 with enzymes capable of inserting or writing sequences of DNA, including entire genes, without the need for double-strand DNA breaks. @Tome_bio’s most advanced PGI technology, called integrase-mediated PGI (I-PGI), utilizes proprietary integrases & is based on groundbreaking PASTE technology first discovered by Tome’s Co-
Founders, @omarabudayyeh & @jgooten - while at @MIT as investigators.
Jan 22 • 15 tweets • 6 min read
1/@PrimeMedicine has issued a corporate update-as part of its #JPM2024 presentation in which $PRME reported: 1)recent business progress 2) its strategic priorities for 2024 3)presentation of the clinical progress it achieved in 2023 #BioTech #CRISPR #GeneEditing #JPM24 🧵👇
2/@PrimeMedicine’s proprietary platform - #PrimeEditing is the only #GeneEditing technology which can edit, correct, insert or even delete large DNA sequences in any target tissue - thus making it the most advanced & promising editing technology with $PRME owing its full rights
Jan 19 • 7 tweets • 3 min read
1/@MeiraGTx announced an asset purchase agreement with @JanssenUS - a @JNJNews company, for its remaining interests in bota-vec for the treatment of XLRP & a technology transfer agreement for bota-vec manufacturing. #BioTech $JNJ #RetinitisPigmentosa #Ophthalmology #Choroideremia
2/#RetinitisPigmentosa is a group of rare genetic eye diseases that damage light-sensitive cells in the retina thus leading to loss of sight. In ~10% of RP cases, the gene is passed from the mother to her children resulting in a form of #RP known as #XLRP.
Jan 16 • 5 tweets • 2 min read
1/@VertexPharma announced today that the @US_FDA has approved CASGEVY (exagamglogene autotemcel [exa-cel]), a #CRISPR/Cas9 #GeneEditing #CellTherapy for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. #BioTech @CRISPRTX $CRSP $VRTX
2/CASGEVYTM is a non-viral,ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break.
Jan 10 • 15 tweets • 6 min read
1/@BeamTx has issued a corporate update - as part of its #JPM2024 presentation in which $BEAM reported: 1)recent business progress 2)highlighted its strategic priorities for 2024 3)presented the clinical progress the company achieved in 2023. #BioTech #CRISPR #GeneEditing #JPM24
2/@BeamTx’s most advanced venture is its Sickle Cell Disease Franchise. By trying to find a long-term treatment - $BEAM has developed a 3 Waves strategy for SCD with the intention to progressively expand the reach of its #BaseEditing platform to broader subsets of SCD patients.
Jan 6 • 11 tweets • 4 min read
1/@intelliatx has issued an update highlighting its strategic priorities & future milestones for 2024. Here’s my 🧵which summarises $NTLA updates of its prioritised pipeline, the progress made in 2023 & its current corporate status. #BioTech #CRISPR #JPM24 #GeneEditing #JPM2024
2/@intelliatx’s 2024-6 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 #GeneEditing programs outside the liver 4. DNA writing
Sep 17, 2023 • 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
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.
Feb 25, 2023 • 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 🧵
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
Feb 23, 2023 • 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
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.👇
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
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
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