TA Ventures’ Post

BlueRock Therapeutics has implanted lab-created neurons into 12 individuals battling Parkinson's. Their aim is to have these cells produce the much-needed dopamine, potentially curbing the debilitating symptoms of the disease. It's fascinating because this experiment stands tall as one of the grandest tests of embryonic-stem-cell technology, an area that has been the center of heated debates for years. While the initial results are promising with some patients showing decreased symptoms and an uptick in dopamine cells, experts suggest treading with caution. There's always the looming shadow of the placebo effect, and more consistent results are a must! See the full article below 🔗 #Parkinsons #LifeSciences #Biotech

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Kyverna's $319M IPO adds to fervor for autoimmune cell therapies: California biotech Kyverna Therapeutics will garner $319 million from its Thursday morning Nasdaq debut as it rides a small, but welcome, wave of investor enthusiasm for initial public offerings. The mid-stage cell therapy maker priced at $22 apiece, selling 14.5 million shares $KYTX. Perhaps in a sign of investor appetite for Kyverna’s work to bring CAR-T cell therapies into the autoimmune world, beyond their initial proving ground in blood cancer, the biotech was able to haul in a larger chunk of capital than it originally expected. Last week, the company set out to raise about $182 million but quickly broadened that ask to $272 million on Tuesday. Kyverna originally sought a price range of $17 to $19 last week but upped it to $20 to $21 earlier this week. It went even higher for the final pricing. Its bigger-than-planned debut follows the suit of three other biotechs to upsize their offerings in the first batch of 2024 IPOs: CG Oncology, ArriVent BioPharma and Alto Neuroscience. Another, cardiometabolic-focused Fractyl Health, did not upsize its offering last week and is the only one so far to trade below its IPO price. Kyverna will quickly be followed by gene editing biotech Metagenomi, which is expected to go public Friday morning and could set the tone for whether public investors are willing to pump money into biotechs that have yet to enter human testing and don’t have line-of-sight to clinical data for multiple quarters or even years. Kyverna, though, fits squarely into the proof-of-concept stage that public investors have favored over the past two years of minimal IPOs. Its listing comes amid multiple clinical studies of its lead autologous CD19 CAR-T for various autoimmune conditions in rheumatology and neurology. Peter Maag “What has been missing in cell therapy is the large indication,” Kyverna CEO Peter Maag told Endpoints News last year. It’s one of more than a dozen cell therapy makers pushing cell therapy into autoimmune diseases. “We’re the large indication.” The biotech still had about $77 million in cash, equivalents and marketable securities at the end of last September, but eventually needed deeper pockets to widen the scope of its lead cell therapy. Dubbed KYV-101, the therapy derives from the National Institutes of Health and is currently in clinical testing for lupus nephritis. In recent months, it’s been cleared for trials in multiple sclerosis, systemic sclerosis and myasthenia gravis, as well. Kyverna is also working with universities like Stanford, the University of Pennsylvania, the University of California San Francisco and the University of Massachusetts for investigator-initiated trials. In Germany, KYV-101 is also going through so-called… #lucidquest #genetherapy #celltherapy

It’s a moment that was nearly two decades in the making: the FDA has approved vamorolone, a steroid-like drug to treat boys with #Duchenne muscular dystrophy. Pioneered on science that began at our Center for Genetic Medicine Research, the drug from ReveraGen BioPharma Inc. will be available in 2024. Read what Dr. Mark Batshaw, former chief academic officer, and Kolaleh Eskandanian, PhD, MBA, PMP, vice president and chief innovation officer, have to say about this new treatment option. #DMD #Innovate4Kids 

Hot off the press: Calidi Biotherapeutics’ Chief Scientific Officer, Antonio F. Santidrian. Ph.D., emphasizes the importance of having a talented team with a vision for commercialization. This is crucial for developing a potent, safe, and efficacious product in the clinic. Calidi Biotherapeutics continues with the task to bring breakthrough immunotherapeutics to the clinic.   Read the article from Biocompare below: #calidi #stemcells #immunotherapy #endcancer

Even with landmark approvals coming late last year for new genetic medicines in sickle cell disease, Beam Therapeutics CEO John Evans is convinced his startup can push the field further. In our latest Endpoints News Slack interview, I chatted with Evans about what success looks like for Beam's own sickle cell programs, the evolving and uncertain business model of paying for one-time genetic cures, and speculating on what CRISPR may look like in two decades, or 2044. My latest:

Gene&Cell Therapy >> Kyverna's $319M IPO adds to fervor for autoimmune cell therapies: California biotech Kyverna Therapeutics will garner $319 million from its Thursday morning Nasdaq debut as it rides a small, but welcome, wave of investor enthusiasm for initial public offerings. The mid-stage cell therapy maker priced at $22 apiece, selling 14.5 million shares $KYTX. Perhaps in a sign of investor appetite for Kyverna’s work to bring CAR-T cell therapies into the autoimmune world, beyond their initial proving ground in blood cancer, the biotech was able to haul in a larger chunk of capital than it originally expected. Last week, the company set out to raise about $182 million but quickly broadened that ask to $272 million on Tuesday. Kyverna originally sought a price range of $17 to $19 last week but upped it to $20 to $21 earlier this week. It went even higher for the final pricing. Its bigger-than-planned debut follows the suit of three other biotechs to upsize their offerings in the first batch of 2024 IPOs: CG Oncology, ArriVent BioPharma and Alto Neuroscience. Another, cardiometabolic-focused Fractyl Health, did not upsize its offering last week and is the only one so far to trade below its IPO price. Kyverna will quickly be followed by gene editing biotech Metagenomi, which is expected to go public Friday morning and could set the tone for whether public investors are willing to pump money into biotechs that have yet to enter human testing and don’t have line-of-sight to clinical data for multiple quarters or even years. Kyverna, though, fits squarely into the proof-of-concept stage that public investors have favored over the past two years of minimal IPOs. Its listing comes amid multiple clinical studies of its lead autologous CD19 CAR-T for various autoimmune conditions in rheumatology and neurology. Peter Maag “What has been missing in cell therapy is the large indication,” Kyverna CEO Peter Maag told Endpoints News last year. It’s one of more than a dozen cell therapy makers pushing cell therapy into autoimmune diseases. “We’re the large indication.” The biotech still had about $77 million in cash, equivalents and marketable securities at the end of last September, but eventually needed deeper pockets to widen the scope of its lead cell therapy. Dubbed KYV-101, the therapy derives from the National Institutes of Health and is currently in clinical testing for lupus nephritis. In recent months, it’s been cleared for trials in multiple sclerosis, systemic sclerosis and myasthenia gravis, as well. Kyverna is also working with universities like Stanford, the University of Pennsylvania, the University of California San Francisco and the University of Massachusetts for investigator-initiated trials. In Germany, KYV-101 is also going through so-called “Individueller Heilversuch,” or one-patient “treatment… #lucidquest #genetherapy #celltherapy

Today Pluri was featured in Genetic Engineering & Biotechnology News's article titled "Improving Cell Therapy Supply Chains". The article previews a presentation Pluri's CTO Lior Raviv will be giving, at the upcoming Advanced Therapies conference in London, focusing on building supply chains for allogeneic cell therapies. Lior Raviv: “We understood regulatory needs from early in process development, so once we broadened our product to different territories, we didn’t have to start from the beginning.” Read more in the link below. #celltherapy #supplychain #biotechnology

OrganoidBase™ now features an expanded range of PDXO models, totaling >400! Boost your studies with free access to a unique collection of patient-relevant organoid models. 🔬 New additions include Adrenal Gland and Uterine models, expanding our offering to 20 different cancers. OrganoidBase also provides you with additional benefits such as: ✅Easy search functionality ✅Cancer genomic profiling data ✅Matched patient/PDX model features ✅Characterization data and visual analysis tools ✅Dose-response curve data and cross-referencing capabilities Get free access ➡️ https://bit.ly/44UMBOX #Organoids #DrugScreening #DrugDiscovery #CancerResearch

It’s great to see this article in Genetic Engineering & Biotechnology News from my colleague Lior Raviv- if you’d like to learn more about Pluri, catch up with us at Advanced Therapies next week in London!

Hi! We're Mongoose Bio - an early-stage clinical company revolutionizing the delivery of T cell precision therapies against common and rare solid tumors. What should you know about our approach? Well, we've developed a unique and disruptive approach to adoptive T cell therapies using epigenetic reprogramming of autologous Tcm cells harboring defined TCR’s against empirically validated highly immunogenic tumor targets to generate active and persistent TCR-T therapeutics. We believe our approach has several distinct advantages: - Reprogramed central memory T cells (Tcm) offer a significant opportunity to minimize toxicity, accommodate outpatient-based treatments and innovative combination strategies; - Reprogramed Tcm are differentiated from TIL and CAR-T products that typically rely on terminally differentiated Teff and Tex cells that have reduced regenerative capacity, are rarely persistent, and only display limited efficacy. Interested? Stay tuned..... www.mongoosebio.com Info@mongoosebio.com