#Science & #Innovation. Special interest in #BioTech, #GeneEditing, #EpiGeneticEditing, #GeneTherapy 🧬 #CRISPR & #Ophthalmology. #Markets & #Stocks 📈
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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.
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. 
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.
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. 
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 
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 
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
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 
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.
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 
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-
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 
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.
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.
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. 
2/@intelliatx’s 2024-6 main Strategic Priorities:
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.
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 
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.👇
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 
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 