The Gentleman from Indiana. BA, MPA, JD.
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ALT WOW! Caribou Biosciences has just presented an updated clinical data from its ongoing CB-011 CaMMouflage phase 1 clinical trial in cancer patients with relapsed or refractory multiple myeloma (r/r MM). The CB-011 CaMMouflage phase 1 clinical data which was presented at the 2026 European Hematology Association Annual Meeting - EHA26, demonstrated deep and durable responses and further supports the potential of CB-011 as a best-in-class allogeneic CAR-T Cell Therapy for second-line relapsed or refractory multiple myeloma patients.
ALT WOW! Intellia has just presented additional positive results from its global Phase 3 HAELO clinical trial of lonvo-z - formerly known as NTLA-2002, which is an in vivo CRISPR gene editing candidate aimed to treat hereditary angioedema (HAE). This new data showed that lonvo-z has met several other key secondary endpoints - besides meeting its primary and key secondary endpoints , including monthly rate of attacks which requires on-demand treatment in weeks 5-28 as well as moderate / severe attacks following treatment of NTLA-2002. This new data continues to support Intellia’s biologics license application (BLA) submission for lonvo-z which was initiated in April with the FDA and the company continues to anticipate regulatory approval and a U.S. launch in the 1H of 2027.
ALT WOW! $NTLA has announced unprecedented positive results from its global Phase 3 HAELO clinical trial of lonvo-z in hereditary angioedema (HAE)! @intelliatx’s Phase 3 HAELO trial of lonvoguran ziclumeran (lonvo-z) met its primary endpoint - a one-time infusion of lonvo-z reduced attacks by 87% versus placebo, with a mean monthly attack rate of 0.26 in the lonvo-z arm compared with 2.10 in the placebo arm during the six-month efficacy evaluation period (weeks 5 to 28). In addition all key secondary endpoints were met (!) and lonvo-z has demonstrated favorable safety and tolerability data! Based on this top tier data Intellia has initiated a rolling BLA submission to the FDA to seek regulatory approval which paves the way for a potential U.S. launch of lonvo-z as soon as the first half of 2027! This is a huge and historic milestone in the CRISPR and Gene Editing field with a valid and undisputed clinical proof that In Vivo CRISPR Gene Editing is truly a “one and done” therapeutic pathway!
ALT WOW! Tune Therapeutics has just presented positive clinical data from its phase 1b/2a trial aimed to evaluated the safety and tolerability of TUNE-401 - a first-in-class Epigenetic silencer for Patients with Chronic Hepatitis B (CHB). Tune’s clinical data demonstrated deep and durable antiviral activity - making it the first clinical evidence of epigenetic silencing in hepatitis B virus (HBV), thus offering a possible new therapeutic pathway for HBV patients.
ALT Here is an excellent article by The Economist which sheds light on a new and promising Gene Editing modality called EpiGenetic Editing. Unlike its older and more clinically mature Gene Editing “siblings” - CRISPR, Base Editing, Prime Editing and RNA Editing, EpiGenetic Editing is just getting underway with several promising companies which are advancing their programs into clinical trials in human patients. Companies such as nchromabio, Tune Therapeutics, Scribe Therapeutics and Epic Bio are all advancing their own clinical programs which deliver a single dose of Epigenetic editors into the patient’s body in order to control the expression of a specific gene which is the root cause of a certain genetic disease. Thus - instead of making a cut in the patient’s DNA in the same way that CRISPR does, EpiGenetic editing hope that by using a less invasive mechanism it would be able to make Gene Editing safer and without any undesirable effects.
ALT HBV causes serious liver infection & increases the risk of health issues like cirrhosis, liver failure & liver Cancer. Chronic HBV infection is characterized by the persistence of covalently closed circular DNA - cccDNA - a unique DNA structure that forms in response to HBV.
ALT This jpmorgan chart wonderfully describes the problematic situation that many big Pharma companies - such as $PFE $LLY $NVO $JNJ $NVS $BMY $AZN $ABBV $MRK $SNY and others, are now facing - the patent cliff scenario. Facing the loss of many of their patents and IP rights, big Pharma companies are currently under an immense pressure on both their pipeline and their future revenue streams. The patent cliff will reach its peak during 2027 & 2028 when many drugs with a total worth of $180B - which is equal to ~ 12% of the entire global market, will go off patent thus leading to massive sale losses. This situation not only puts big Pharma companies under a massive pressure on both their pipeline and their future revenue streams but forces them to initiate M&A deals in an unprecedented scale in order to compensate for this potential loss.
ALT The driving force behind the recent “M&A frenzy” in the BioTech and Pharma ecosystem is the patent cliff scenario which placed many big Pharma companies in a very problematic situation. The fact that by 2030 many prescription drugs - which hold a commercial potential to generate more than $300B in combined revenue, will face patent expiry is forcing Big Pharma companies to aggressively replenish their pipelines - mostly via M&A deals with smaller BioTech companies. According to this excellent chart by J.P. Morgan - Q1 of 2026 has been super strong M&A-wise and it seems that big Pharma companies are definitely positioning themselves to replenish their clinical pipelines and to generate new growth engines.
ALT SparingVision - a clinical-stage genomic medicine company developing vision-saving treatments for ocular diseases, today announces that it has selected a second target as part of its strategic collaboration with Intellia Therapeutics to develop novel genomic medicines utilizing CRISPR-based gene editing technologies for the treatment of ocular diseases.
