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
3 $GRPH gene correction platform involves editing hematopoietic #stem cells found in the #bonemarrow that develop into various types of #blood#cells. Since red blood cells lose their genomic DNA during maturation-tracking #GeneEditing in mature cells via sequencing is impossible
4/The problem of the inability to mesure the efficacy of #GeneEditing in #SCD patients was solved by using immature red blood cells called #reticulocytes which retain their RNA and can be sequenced in order to assess the #gene correction levels after using nula-cel. #ASH22
5/Based on this knowledge, $GRPH sought to develop a single-cell RNA sequencing method that could measure #GeneEditing outcomes in reticulocytes. It first measured the genetic makeup of reticulocytes from healthy donors-AA, people with SCD trait-AS, & with #sicklecelldisease-SS.
6/Results from both experiments demonstrated the single-cell RNA sequencing method’s ability to precisely and reproducibly measure and differentiate the healthy donors - AA, people with SCD trait - AS and #SickleCellDisease patients - SS. #ASH22
1/🚨WOW! A huge earthquake in the CRISPR & Gene Editing field happened last night after $EDIT has announced that the U.S Federal Court of Appeals has vacated (!) the Patent Trial & Appeal Board’s (PTAB’s) previous decision🧵👇which granted all of the rights for CRISPR/Cas9 Gene Editing to the Broad institute and ruled against University of California, the University of Vienna & Nobel Prize winner Emmanuelle Charpentier - the co-inventor of CRISPR Cas9 and the co-founder of CRISPR Therapeutics. $CRSP $NTLA $XBI
2/In February of 2022 the U.S. Patent & Trademark Office has issued a crucial decision in favour of the Broad Institute, which validated its patents for CRISPR/Cas9 Gene Editing in human cells. This provided $EDIT with strong IP rights & gave it a huge commercial advantage. $XBI
3/But the losing side in this dispute over the patents for CRISPR/Cas9 editing in human cells - the University of California, the University of Vienna & Emmanuelle Charpentier - the co-founder of $CRSP refused to accept this ruling and instead filed an appeal.
1/Here’s an excellent article - published in the recent @WIRED issue, by Nobel prize winner Jennifer Doudna about how AI & machine learning are amplifying the impact of CRISPR & Gene editing in all walks of life - medicine, agriculture, climate change & research landscape.🧵👇
2/Jennifer Doudna is an American biochemist who discovered CRISPR Cas9 as a Gene Editing tool & had received the 2020 Nobel Prize in Chemistry with Emmanuelle Charpentier for their discovery. She also founded several BioTech companies like $NTLA, $CRBU, Scribe, Mammoth & others.
3/According to Jennifer Doudna - 2025 will be a pivotal year in which the ever growing use of Artificial Intelligence and machine learning will amplify the effects and reach of CRISPR and Gene Editing in many aspects of our lives - curative medicines, agriculture & lab research.
1/🚨WOW! $SANA announced initial positive results from its first-in-human study of UP421 - an allogeneic primary islet Cell Therapy engineered with Sana’s proprietary hypoimmune (HIP) technology for patients with type 1 Diabetes, without the use of any immunosuppression $XBI 🧵👇
2/Type 1 diabetes is a chronic (life-long) autoimmune disease that prevents the patient’s pancreas from making Insulin - an important hormone that regulates the amount of glucose (sugar) in the blood. Type 1 diabetes affects both children and adults & requires daily management with insulin injections and blood sugar monitoring.
3/Type 1 Diabetes affects roughly 8.4M people worldwide. The number of patients is constantly rising and the prevalence of type 1 diabetes is expected to double over the next decade. Interestingly enough almost 80% of T1D patients are from high-income countries with insurance.
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 🧵👇
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.
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
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
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
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
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.
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