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
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.
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.
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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1/@PrimeMedicine’s recent #JPM26 presentation emphasised its strategic priorities & the company’s planned milestones for 2026-7. Here’s my🧵👇which focuses on PRIME’s new prioritised pipeline, the progress $PRME has made during 2025 & its current corporate status. $XBI #JPM2026
2/@PrimeMedicine’s proprietary platform - Prime Editing is the only Gene Editing platform which can edit, correct, insert or even delete large DNA sequences in any target tissue. This makes it the most advanced & promising editing technology with $PRME owing its full IP rights.
3/IMO the most important key takeaway from @primemedicine’s #JPM26 presentation is that in 2026 $PRME intends to submit INDs and to initiate clinical trails for both of its key programs - PM577 for Wilson’s disease and PM647 for AATD. Moving forward with its liver franchise and providing positive human clinical data are a threshold for $PRME to enter into a significant collaboration with a big Pharma company.
1/🚨WOW! According to this excellent Economist’s article 🧵👇 it seems that the 🇬🇧 big Pharma company AstraZeneca - one of Europe’s and England’s last remaining BioTech and Pharma moguls, is moving its business away from England! $AZN leaving the UK is a huge negative milestone in the deterioration process of both the British and the European BioTech ecosystems which are rapidly collapsing and losing ground to the American BioTech ecosystem - following President Trump’s Most-Favored-Nation (MFN) policy as well as to the Chinese one which has made an exponential progress. $XBI
2/The British government has always considered its life science industry as one of the Crown Jewels of its economy and rightfully so - a 145 billion dollars industry which employs over 300,000 high skilled employees is crucial for the British economy. The importance of the Pharma and Biotech industry has led the British government to a present a new governmental initiative to support the industry and that was introduced by the Labor government just 3 months ago.
3/But it seems that the British BioTech & Pharma sector is about to face its biggest challenge ever after AstraZeneca - Britain’s biggest and leading Pharma company is shifting its corporate strategy including a possible relocation of $AZN entire operations to the U.S. including mass investments being made outside England!
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