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Gene&Cell Therapy >> Gilead partners with AI startup Genesis Therapeutics to make small molecule drugs: A well-funded California biotech startup using AI to design and improve small molecule drug candidates has just struck its third pharma partnership in four years. Gilead announced a new preclinical research deal with Genesis Therapeutics on Tuesday morning. Genesis, which raised $200 million from investors a year ago, will receive $35 million upfront across three drug targets picked by Gilead, plus undisclosed milestone payments if Gilead takes the drugs into clinical development. The startup previously partnered with Genentech in 2020 and Eli Lilly in 2022. Evan Feinberg, Genesis’ co-founder and CEO, told Endpoints News in an email that he couldn’t disclose further details about the kinds of diseases or drug targets the company is working on in any of its pharma partnerships. Gilead is best known for its development of antiviral drugs for hepatitis and HIV, and its CAR-T cell therapy for cancer. While it has dabbled in using AI for development of clinical biomarkers, the Genesis partnership is its first major deal focused on using AI to design drugs. Genesis launched with a small seed round in 2019, has since raised more than $300 million total, and has “multiple years of runway,” according to Feinberg. The startup is based around two key AI studies from Stanford University. One described a neural network dubbed PotentialNet to help optimize binding of small molecules to proteins. The second study used AI to predict the absorption, distribution, metabolism, and excretion characteristics of molecules — so called ADME properties — which could help drugmakers pick promising compounds to advance into animal and human studies, and avoid potentially problematic ones. Genesis has remained quiet about how it’s improved these AI models over the past five years, but has branded its AI platform as GEMS, short for Genesis Exploration of Molecular Space. “GEMS employs ADME-conditioned language models for molecular generation, diffusion models for protein-ligand docking predictions, and physical machine learning methods for potency prediction,” Feinberg said. He added that GEMS is especially good with “difficult-to-drug targets” and optimizing several parameters of a molecule at once. The company’s lead program, still in preclinical development, is an inhibitor of PIK3CA, an enzyme with multiple mutations that are linked to cancer. Feinberg said that Genesis is using AI to create a drug that inhibits a range of these mutant proteins while avoiding toxicities from targeting healthy versions of the protein. #lucidquest #genetherapy #celltherapy

🕺 DISCO Pharmaceuticals GmbH launches with €20 Million Seed Financing! 🚀 🔬 Based out of Germany & Switzerland, DISCO are unravelling the surfaceome of cancer cells on a large scale, uncovering novel targets and pioneering the development of ground-breaking, first-in-class drugs. 💸 The financing is backed by a prominent investor syndicate, which includes Panakes Partners, M Ventures , AbbVie Ventures, with Sofinnova Partners spearheading the financing. 🗣 "𝘞𝘦 𝘣𝘦𝘭𝘪𝘦𝘷𝘦 𝘵𝘩𝘢𝘵 𝘰𝘶𝘳 𝘴𝘶𝘳𝘧𝘢𝘤𝘦𝘰𝘮𝘦 𝘥𝘪𝘴𝘤𝘰𝘷𝘦𝘳𝘺 𝘵𝘦𝘤𝘩𝘯𝘰𝘭𝘰𝘨𝘺 𝘪𝘴 𝘵𝘳𝘶𝘭𝘺 𝘥𝘪𝘴𝘳𝘶𝘱𝘵𝘪𝘷𝘦 𝘢𝘯𝘥 𝘸𝘪𝘭𝘭 𝘵𝘳𝘢𝘯𝘴𝘧𝘰𝘳𝘮 𝘰𝘯𝘤𝘰𝘭𝘰𝘨𝘺 𝘵𝘳𝘦𝘢𝘵𝘮𝘦𝘯𝘵 𝘰𝘱𝘵𝘪𝘰𝘯𝘴 𝘢𝘯𝘥 𝘶𝘭𝘵𝘪𝘮𝘢𝘵𝘦𝘭𝘺 𝘪𝘮𝘱𝘳𝘰𝘷𝘦 𝘰𝘶𝘵𝘤𝘰𝘮𝘦𝘴 𝘧𝘰𝘳 𝘱𝘢𝘵𝘪𝘦𝘯𝘵𝘴." Roman Thomas ▶ 2024 has kicked off in excellent fashion, long may the good news continue! Check out the link posted below to learn more about DISCO Pharmaceuticals GmbH - congratulations to all involved. #biotech #drugdiscovery #proteomics #surfaceome

Will 2024 Be The Year of Artificial Intelligence in Drug Discovery? The London-based Isomorphic Labs, a unit of Google’s parent company Alphabet company, announced it is entering into two strategic research collaborations — one with Elli Lilly and one with Novartis — to discover small molecule therapeutics for multiple targets. The two partnerships, potentially worth a combined $3 billion, begin with the startup receiving upfront payment of $45 million from Eli Lilly, and as much as $1.7 billion to be paid according to performance-based milestones, while Novartis, has agreed to pay $37.5 million upfront, and an additional $1.2 billion in additional incentives going forward. #ai #artificialintelligence #drugdiscovery #drugdelivery #medicalsciences #pharmaceutics #medicine #oncology #cancer #genetherapy Google Novartis Eli Lilly and Company Insilico Medicine

🚀 Exciting times in the world of therapeutics! We've published a stunning new article that explores some of the most influential therapeutics startups in the hub of biotech innovation - Cambridge, Massachusetts. Dive in to learn about these groundbreaking startups and the stalwarts who are leading them. 🌍Read it here: https://lnkd.in/eG5Z7Qz6 Featuring: Orna Therapeutics, Remix Therapeutics, Immunitas Therapeutics, founded by Amanda Wagner, Vesigen Therapeutics, Inc., Mediar Therapeutics, founded by Paul Yaworsky Civetta Therapeutics, PineTree Therapeutics Inc Carmine Therapeutics, founded by XiangQian (XQ) Lin InnoVoyce, Vedere Bio II NextPoint Therapeutics, Inc., founded by Gordon Freeman and Xingxing Zang MOMA Therapeutics, Vigil Neuroscience Bicara Therapeutics Cambridge is proving to be a melting pot of scientific innovation, fostered by these promising therapeutics startups and their remarkable founders. Here's moving closer to the day when disease and illness will be things of the past! #Biotech #Startups #Innovation #Therapeutics #CambridgeStartups

Boosting a novel delivery platform for RNA therapeutics: ETH Zürich spin-off Procavea Biotech receives the CHF 150,000 Venture Kick funding. Congrats and best of success in the new year and beyond - cheers for the team of innovators Michael Wetter, Tom Edwardson, Richard Quaderer, and Donald Hilvert! https://lnkd.in/euA-r2_W About: Procavea Biotech is on a mission to revolutionize the delivery of RNA therapeutics and small molecule drugs. The company brings a first-in-class protein-based delivery platform for oligonucleotides and small molecules to the market. The technology was developed by at ETHZ and is protected in two patents. The current focus is on the Antisense and RNAi Therapeutics market, where there is great need for innovative delivery solutions. Procavea's target customers are pharmaceutical companies who will gain a competitive advantage by using our unique technology. Through strategic partnerships the company will license out bespoke delivery systems at various stages of development to meet its customers' needs. Long term Procavea Biotech aims to develop its own therapeutic pipeline. #VentureKick #SwissStartups

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Ivan Cheung's post-Eisai life takes hold at NextPoint Therapeutics; Sanofi, Takeda announce CFO exits: Ivan Cheung Ivan Cheung, who led Eisai’s US business and was a central figure in the biotech’s Alzheimer’s work, has stepped into the CEO position at a Cambridge, MA startup. He started running immuno-oncology outfit NextPoint Therapeutics last month, having departed Eisai shortly after Eisai secured full FDA approval for its Alzheimer’s treatment Leqembi last summer. The 30-employee upstart has reeled in backing from Big Pharmas like Sanofi and Bayer, as well as VC firms like MPM Capital and Catalio Capital. Cheung spent nearly 20 years across multiple units of Tokyo-based Eisai, including in oncology with the cancer drug Lenvima. But the work on Leqembi, with partner Biogen, attracted the spotlight in recent years as Alzheimer’s sprinted to the fore, becoming one of the next big frontiers in biopharma R&D after years and years of disappointing clinical setbacks. “If you look back at my career, at my previous role, whether it’s Lenvima or Leqembi, it’s really finding that mechanistically unique asset that can really move the needle,” Cheung said in an interview with Endpoints News. “NextPoint, to me, is a very natural progression to do the same thing again. In life, you’re fortunate enough to do it once, maybe twice,” Cheung said. “And I hope three times.” NextPoint is going after a new immunotherapy target with the HHLA2 checkpoint axis, and its work is based on research out of Albert Einstein College of Medicine professor XingXing Zang and Dana-Farber Cancer Institute researcher Gordon Freeman. Cheung noted there are a “good number of patients who still don’t benefit from the PD-1/PD-L1 therapy.” The company entered the clinic last August and is in the process of preparing to dose the first patient in a clinical trial of its second cancer therapeutic candidate, Cheung said. An $80 million Series B, disclosed last January, will carry NextPoint through the two Phase I studies, Cheung said. As for further financing, Cheung said he’s “not gonna take money unless I exactly know what I’m going to use that for, but the investor appetite is there for sure.” “I like things efficient. I like things agile. I’m always of the thought that a small, strong team can beat bigger competitors,” Cheung said. NextPoint brought on immuno-oncology pioneer Leena Gandhi as chief medical officer last winter, and its board includes ex-Scorpion Therapeutics CEO Axel Hoos. — Kyle LaHucik Jean-Baptiste Chasseloup de Chatillon → Jean-Baptiste Chasseloup de Chatillon has worked side by side with Paul Hudson as CFO of Sanofi since the chief executive’s arrival in 2019. But de Chatillon is leaving the biopharma world completely by April 1 to replace Nicolas… #lucidquest #genetherapy #celltherapy

🚀 AI Drug Discovery Weekly Wrap-Up 🚀 Here's a quick rundown of this week's key highlights: 📈 Xtalpi’s Stock Soars After Major Deals Shenzhen-based Xtalpi saw its shares double in value, boosted by a 28% rise in revenue and a $135M partnership with GCL Group. Despite widening losses, Xtalpi continues to invest heavily in AI-powered drug discovery, attracting big names like Pfizer, Google, and SoftBank. Source: https://lnkd.in/eH6pnTm3 🤝 Eli Lilly Partners with Genetic Leap in $409M AI Drug Deal Eli Lilly continues to deepen its RNA research with a $409M deal with Genetic Leap, a biotech specializing in AI models for RNA-targeted drugs. The partnership aims to explore new genetic drug candidates in high-priority areas like neurodegeneration and diabetes. Source: https://lnkd.in/gnqF9gRY 💡 Noetik Raises $40M to Revolutionize Cancer Treatment with AI Noetik, an AI startup from the Bay Area, secured $40M in Series A funding to expand its innovative cancer research platform. Using its OCTO AI model, Noetik aims to better match cancer patients with effective treatments by diving deep into tumour biology. Source: https://lnkd.in/dPmvtn_j 🧪 🧬 Recursion's AI-Driven Growth Recursion aims to release data on seven pipeline candidates in 18 months, possibly ten with Exscientia's merger. Recent Phase II results for CCM candidate REC-994 showed promise but disappointed investors due to limited patient-reported outcomes. CEO Chris Gibson highlights Recursion’s broad AI-driven approach and plans to develop 100 drug candidates in the next decade. Source: https://lnkd.in/eQ2QEC93 For even more AI-related topics, take a look at the full AI Driven Drug Discovery Summit (November 12-14, Boston) program here 👉 https://lnkd.in/emjPGCBi #AIDDD #aidrivendrugdiscovery #aidrugdiscovery

Recent Funding: New #AI drug discovery powerhouse Xaira Therapeutics (San Francisco) rises with $1 billion in funding - the largest initial funding commitment in ARCH history. Incubated by ARCH Venture Partners and Foresite Labs with none other than Marc Tessier-Lavigne, Ph.D., former CSO at Genentech, at the helm. The company was co-founded by David Baker, Ph.D., professor of biochemistry and director of the Institute for Protein Design at the University of Washington. Xaira will combine machine learning, data generation and therapeutic product development to build a platform for #drugdiscovery. Endeavor BioMedicines (San Diego) pulls in $132M to back #cancer, lung disease drugs. The oversubscribed series C stretches the company's cash runway through 2026. Money will be channeled into lead asset ENV-101, which is designed to block a cellular wound-healing pathway known as Hedgehog. Phase 2a data set to be presented at the American Thoracic Society annual meeting show that no patients treated with ENV-101 had signs of disease progression, compared to two given placebo. Norwest Venture Partners (Menlo Park) announces $3 Billion global fund to partner with the next generation of enduring companies. Norwest expanded the focus of its healthcare practice over the last two years by adding #biotech. The most recent biotech company investments include Engrail Therapeutics and Ray Therapeutics, Inc. Recent Layoffs: Bristol Myers Squibb plans to cut costs by $1.5 billion by the end of 2025 in a massive restructure that includes laying off about 2,200 employees. The company is shuttering its Cancer Immunology & Cell Therapy Thematic Research Center in Redwood City, CA. M&A, Deals, Partnerships: Incyte announced that it is buying Escient Pharmaceuticals for $750 million to gain control of a pipeline led by clinical-phase treatments of atopic #dermatitis and other skin conditions. Escient’s lead asset is an antagonist of Mas-related G protein-coupled receptor X2 (MRGPRX2), a receptor that is expressed on mast cells. The small molecule, EP262, is in Phase Ib/II trials in those three conditions and clinical proof-of-concept data are due early in 2025. Cidara Therapeutics (San Diego) reacquires global development and commercial rights to CD388 and announces private placement financing of $240 Million. The financing was led by RA Capital Management to fund Phase 2b clinical trial of CD388, which is active against all strains of influenza A and B, and being developed for pre-exposure prophylactic treatment. Regeneron expands in gene editing with $100 million #CRISPR drug developer Mammoth Biosciences deal (Brisbane, CA). Through their collaboration, the companies plan to develop in vivo gene therapies that combine Regeneron’s viral vector technology with Mammoth’s #DNA editing platforms, which are designed to be more compact than the industry standard.

Yesterday, Merck acquired Caraway Therapeutics, Inc., a company developing small molecule modulators of lysosome function for $610 M including milestone payments. 💲The company had raised $23 M through grants and a Series A co-led by MRL Ventures Fund (Merck) and AbbVie Biotech Ventures, joined by other corporate venture capital (CVC) participants such as Amgen Ventures, and backed by the Michael J. Fox Foundation, Dementia Discovery Fund, and Mayo Clinic, in addition to IVCs. 1️⃣ Caraway’s patient-derived iPSC Core enables reconstitution of disease context-specific neurons to design and test drugs 𝘪𝘯 𝘷𝘪𝘵𝘳𝘰, leveraging their advanced imaging and electrophysiology platform. The lead candidate stimulates the ion channel TRPML1, which regulates cationic flow and enzymatic activity in the essential waste clearing organelle, the lysosome; drives autophagy-lysosomal pathway transcriptional regulation; and maintains interactions between the lysosome and plasma membrane. 2️⃣ TRPML1 activation also drives expression of glucocerebrosidase (GCase), a ‘housekeeping’ enzyme whose deficiency is the causative factor in the lysosomal storage disorder (LSD), Gaucher disease, a focus indication in gene therapy (AVROBIO; Freeline). GCase is encoded by the 𝘎𝘉𝘈 gene and breaks down glucocerebroside in the lysosome. Almost 400 𝘎𝘉𝘈 variants are responsible for the three clinical subtypes of Gaucher arising due to aberrant glycolipid accumulation. Mutations in 𝘛𝘙𝘗𝘔𝘓1 itself are associated with Morquio syndrome (MPS IV) and a wide array of other rare monogenic LSDs.   🧠 Only recently has Parkinson’s disease (PD) been conceptualized as having genetic components, including 𝘚𝘕𝘊𝘈 and 𝘔𝘈𝘗𝘛. Mutations in the 𝘓𝘙𝘙𝘒2 gene have attracted drug development efforts, including via PROTAC (Arvinas) and small molecule (Denali Therapeutics / Biogen) to correct disruptions in cytoskeletal and neurite function, and notably, vesicular trafficking and autophagy, which correlate with LRRK2’s interaction with synaptic α-synuclein, tau aggregation, and inflammation-producing microglia. The most frequent ‘large-effect’ genetic risk factor in PD, however is mutation of 𝘎𝘉𝘈, associated with the aforementioned pathogenic errors in lysosomal flux and ER stress that give rise to toxic macromolecule accumulation characterized as GBA-Parkinson’s. Caraway’s drugs targeting TRPML1 and the related channel TMEM175 across LSDs, GBA-PD (Phase I initiation expected mid-2024), ALS, as well as polycystic kidney disease represent a novel approach, but one familiar to the #longevity community ➡ see link in comments re: disabled macroautophagy as a hallmark of aging. Related: the NLRP3 inflammasome as an increasingly important target across TAs (Roche; Ventus Therapeutics) ♨ #autophagy #raredisease #neurodegeneration #inflammation 🔅 To learn more about the innovative biopharma landscape, portfolio planning, and valuation, please contact me or Adrian Rubstein 🔅

💸 Biotech & pharma fundraising highlights from the past week include 3 IPOs, 2 upsized public offerings, and 3 $100M+ private placements. These are the deals that made news: 👇 Feb 4 – Feb 11, 2024: ➡️ IPO ⬅️ 🔬 Kyverna Therapeutics (NASDAQ: KYTX) priced its upsized IPO, expecting to raise over $319M. Kyverna focuses on cell therapies for autoimmune diseases and is advancing KYV-101 for multiple sclerosis and lupus nephritis.   🧬 Metagenomi (NASDAQ: MGX) priced its IPO at $15 and expects to raise around $93.75M. The company, leveraging a metagenomics-derived toolbox, is focused on developing curative therapeutics through gene editing.   🌟 Telomir Pharmaceuticals, Inc. priced its IPO at $7/share, aiming to raise about $7M. Focused on age reversal, its lead product, TELOMIR-1, targets telomere lengthening for conditions including hemochromatosis and is entering pre-clinical development. 🧠 Alto Neuroscience closed its upsized IPO, raising roughly $147.9M. ➡️ PUBLIC ⬅️ 🦠 4D Molecular Therapeutics (NASDAQ: FDMT) priced an upsized $300M public offering at $29.50/share. 4D is pioneering gene therapies in ophthalmology and pulmonology using its Therapeutic Vector Evolution platform. 🧫 Sana Biotechnology, Inc. priced an upsized public offering, expecting to raise approximately $165M. 👁️ Adverum Biotechnologies completed a $127.5M private placement. The clinical-stage biotech focuses on ocular gene therapies, notably Ixo-vec for wet age-related macular degeneration. 🤫 Silence Therapeutics plc secured $120M through an oversubscribed private placement. The biotech focuses on RNAi therapeutics, with lead candidates zerlasiran (SLN360)for cardiovascular risks and divesiran (SLN124) for conditions such as polycythemia vera. 💊 Mineralys Therapeutics, Inc. secured $120M in a private placement led by TCGX and RA Capital Management. The funds will support R&D of lorundrostat, their lead candidate for cardiorenal diseases related to high aldosterone. ➡️ PRIVATE ⬅️ 🧠 Neurona Therapeutics completed a $120M equity financing led by Viking Global Investors and Cormorant Asset Management, LP. Funds will advance their regenerative cell therapy NRTX-1001 for epilepsy and Alzheimer's, alongside other neural cell therapies for neurological disorders.