Elan Lutinger PharmD, MBA’s Post

Gene&Cell Therapy >> Caribou to start Phase 1 autoimmune trial of CAR-T therapy: Just like other cell therapy companies, Caribou Biosciences is entering the autoimmune disease field. The Berkeley, CA-based biotech disclosed Thursday afternoon that the FDA cleared it to begin a clinical trial of its off-the-shelf cell therapy in lupus, and Caribou expects to start the Phase 1 study by the end of 2024. Caribou is joining the herd of companies studying cell therapy for autoimmune conditions, where interest has exploded after an academic research group led by Georg Schett from the Friedrich-Alexander University Erlangen-Nürnberg reported striking results in a small group of patients with lupus and other autoimmune disorders. Other companies that have entered into the autoimmune cell therapy field recently include Nkarta and CRISPR Therapeutics. Caribou plans to use its off-the-shelf CD-19-directed CAR-T cell therapy in its autoimmune study. The biotech is already testing the treatment, dubbed CB-010, in B cell non-Hodgkin lymphoma where it reported early data in July. The company is planning for a Phase 3 study of CB-010 by the end of the year in non-Hodgkin lymphoma. The therapy is engineered via CRISPR with a PD-1 knockout, which Caribou believes will make the treatment more durable compared to other off-the-shelf attempts. CEO Rachel Haurwitz told Endpoints News ahead of the announcement that Caribou plans to enroll at least 20 patients in its autoimmune study across two cohorts — one with lupus nephritis patients and one with extrarenal lupus patients. The biotech will also be adopting partial HLA matching in its studies of CB-010 between donor sources and the patients it enrolls, a decision it made based on data from the lymphoma study that it believes will improve clinical outcomes. On whether the partial HLA matching will impact how readily ‘off-the-shelf’ the cell therapy will be, Haurwitz said “it does not slow down how patients access therapy. We are able to determine the patient’s HLA type very early in the screening process for either of these trials. And then we simply use that information to select the most appropriate dose for them based on the inventory that we have on hand, so it does not in any way impact the timelines.” Caribou expects to share updated data on its lymphoma study of CB-010 in the coming months. “We look to the company’s 2Q update for evidence of differentiation in oncology and how this ‘look before you leap’ approach positions the company in a competitive development landscape,” wrote Leerink Partners analyst Mani Foroohar in a note Thursday. #lucidquest #genetherapy #celltherapy

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Caribou to start Phase 1 autoimmune trial of CAR-T therapy: Just like other cell therapy companies, Caribou Biosciences is entering the autoimmune disease field. The Berkeley, CA-based biotech disclosed Thursday afternoon that the FDA cleared it to begin a clinical trial of its off-the-shelf cell therapy in lupus, and Caribou expects to start the Phase 1 study by the end of 2024. Caribou is joining the herd of companies studying cell therapy for autoimmune conditions, where interest has exploded after an academic research group led by Georg Schett from the Friedrich-Alexander University Erlangen-Nürnberg reported striking results in a small group of patients with lupus and other autoimmune disorders. Other companies that have entered into the autoimmune cell therapy field recently include Nkarta and CRISPR Therapeutics. Caribou plans to use its off-the-shelf CD-19-directed CAR-T cell therapy in its autoimmune study. The biotech is already testing the treatment, dubbed CB-010, in B cell non-Hodgkin lymphoma where it reported early data in July. The company is planning for a Phase 3 study of CB-010 by the end of the year in non-Hodgkin lymphoma. The therapy is engineered via CRISPR with a PD-1 knockout, which Caribou believes will make the treatment more durable compared to other off-the-shelf attempts. CEO Rachel Haurwitz told Endpoints News ahead of the announcement that Caribou plans to enroll at least 20 patients in its autoimmune study across two cohorts — one with lupus nephritis patients and one with extrarenal lupus patients. The biotech will also be adopting partial HLA matching in its studies of CB-010 between donor sources and the patients it enrolls, a decision it made based on data from the lymphoma study that it believes will improve clinical outcomes. On whether the partial HLA matching will impact how readily ‘off-the-shelf’ the cell therapy will be, Haurwitz said “it does not slow down how patients access therapy. We are able to determine the patient’s HLA type very early in the screening process for either of these trials. And then we simply use that information to select the most appropriate dose for them based on the inventory that we have on hand, so it does not in any way impact the timelines.” Caribou expects to share updated data on its lymphoma study of CB-010 in the coming months. “We look to the company’s 2Q update for evidence of differentiation in oncology and how this ‘look before you leap’ approach positions the company in a competitive development landscape,” wrote Leerink Partners analyst Mani Foroohar in a note Thursday. #lucidquest #genetherapy #celltherapy

🚀 Pharma Industry Breakthroughs to Watch in 2024! 🌟 Weight-loss Revolution: Pharmaceutical giants like Novo Nordisk and Lilly are leading the charge with new oral obesity treatments showing over 15% weight loss in trials! This could redefine the future of non-invasive treatments for obesity. 🌿 💊 Cardiovascular Disease Innovations: New molecular targets and AI-powered diagnostics are pushing the boundaries in personalized heart care. 🔬 Gene Therapy & mRNA Tech: From cancer to rare diseases, CRISPR-based therapies are gaining ground, with the FDA approving groundbreaking treatments in 2023! 2024 is looking transformative for patient care! 🚑 #PharmaTrends #HealthcareInnovation #Pharmaceuticals

Cenicriviroc, initially developed as an anti-HIV drug, inhibits the infiltration and activation of CCR2+/CCR5+ monocytes and macrophages to the site of liver injury, preventing liver fibrosis. In vitro studies led by Dr Amit Awasthi @BRIC- Translational Health Science and Technology Institute (THSTI) showed that Cenicriviroc inhibits Th1-, Th2-, and Th17-cell differentiation while promoting the generation of type 1 regulatory T cells (Tr1), known for preventing inflammation through induction of IL-10. This study is the first to report that Cenicriviroc promotes Tr1 cell generation by up regulating the signature of Tr1 cell transcription factors. Cenicriviroc displayed a protective effect in experimental colitis models by preventing body weight loss and intestinal inflammation and preserving epithelial barrier integrity and thus emerge as a potential therapeutic in controlling tissue inflammation by inhibiting the generation and functions of effector T cells and promoting the induction of anti-inflammatory Tr1 cells. Read More at: 10.1002/eji.202350847 Rajesh Gokhale Department of Biotechnology Translational Health Science and Technology Institute (THSTI)

🚨 FDA's Crucial Week Ahead 🚨 The FDA is set for a pivotal week with four significant target action dates on the horizon. Here’s what’s on the line: 🔹 Roche's Lunsumio: A potential label expansion for this bispecific antibody. 🔹 Rocket Pharmaceuticals: An investigational gene therapy for Danon disease, a rare pediatric heart condition. 🔹 Merck’s MK-2060: Awaiting approval for an oral PCSK9 inhibitor to treat hypercholesterolemia. 🔹 Verona Pharma’s ensifentrine: A novel dual inhibitor being considered for chronic obstructive pulmonary disease (COPD). These decisions could reshape treatments for various conditions and are being closely watched by healthcare professionals, patients, and investors. Stay tuned as the FDA's impending actions promise to make waves in the biotech and pharma landscape. Read the full article at https://lnkd.in/dqNdhy7

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> In latest turn to autoimmune therapies, Cullinan nabs $280M to join lupus space: At Cullinan, immunology is quite literally the new oncology. The biotech announced Tuesday that it’s expanding its pipeline from cancer to include immunology and changing its name from Cullinan Oncology to Cullinan Therapeutics. By doing so, the company is joining the autoimmune craze, becoming the latest biotech to either add or pivot toward a program testing groundbreaking cancer treatments in lupus. The company also closed a $280 million PIPE financing to support the change, with the funding expected to extend its cash runway into 2028. Investors applauded the move, sending Cullinan shares $CGEM up nearly 15% on Tuesday morning. Unlike some competitors, Cullinan isn’t embarking on a full redirection toward autoimmune cell therapy. Rather, it’s positioning systemic lupus erythematosus (SLE) as the new and only indication that it’s pursuing for a previously announced T cell engager program, known as CLN-978. The rest of its pipeline remains unaffected, CEO Nadim Ahmed told Endpoints News in an interview. The excitement about CAR-T’s potential in autoimmune disorders started in 2022, when a German team led by Georg Schett at the University Hospital Erlangen showed that all five lupus patients treated with a CD19-targeting CAR-T therapy went into remission. And it’s only grown since, when the same researchers published follow-up data that revealed 15 patients saw substantially improved symptoms, with eight seeing their disease clear entirely. Cullinan had previously been testing CLN-978 in B cell non-Hodgkin lymphoma, but Ahmed said moving that program forward would have been too costly to know if it might be competitive. “Like most people in the world, we saw the data from Schett and colleagues,” Ahmed said. “We came to the determination that, in lymphoma, it’s going to be really hard to find a development path moving forward.” There are now roughly two dozen companies studying their own cell therapies in autoimmune conditions, according to Mizuho analyst Salim Syed. Bristol Myers Squibb, Novartis, Gilead, CRISPR Therapeutics, Kyverna and Sana are just a few of the bigger names that have entered the market. It’s not just CAR-T either, as Nkarta — a CAR-NK therapy developer — also pivoted toward autoimmune diseases last month, touting better safety as a differentiating factor. Cullinan’s CLN-978 is a CD19xCD3 T cell engager that execs believe can treat a wide variety of B cell mediated autoimmune indications. The company hopes to separate itself from the increasingly crowded market by noting CLN-978’s off-the-shelf nature and emphasizing it can be repeatedly dosed, stressing the advantages of a T cell engager over cell therapy. “If a patient with… #lucidquest #genetherapy #celltherapy

These truly are exciting times in the field of medicine! Although I couldn't attend in-person, I can clearly sense the optimistic vibes for the industry as I read through various publications, tracking the developments this week at the #jpm2024 healthcare conference. My big takeaways: 1. Expect M&A activity to accelerate in 2024; a few focus areas beyond oncology include CNS, Cardiovascular & metabolism including Obesity, Immunology 2. Commercial execution is going to be critical in this decade given robust pipelines across major players and a series of upcoming launches 3. AI, Gen AI and the rise of Digital Biology! Expect to see more partnerships and investments. Promises are extremely strong but drug development is a tricky space. 4. Drug pricing, IRA and the PBM reform. IRA will continue to pressure drug pricing outlook. Wait and watch for the turns the ongoing PBM legislations will take. 5. Cell and gene therapy market set to grow but watch out for the infrastructure and pricing challenges. Seven CGTs got approved in 2023 and this year could see 17!!! But is the health care ecosystem prepared for this influx? #jpm2024 #jpm

𝐋𝐚𝐭𝐞𝐬𝐭 𝐁𝐢𝐨𝐩𝐡𝐚𝐫𝐦𝐚 𝐍𝐞𝐰𝐬 👇 👉 Sarepta Therapeutics achieves full approval and expands labels for its DMD Gene Therapy. 🤝 Roche collaborates with Ascidian Therapeutics to develop gene therapies. 👏 Samsung Biologics and Baxter International Inc. strike a manufacturing deal worth $223 million. ✅ Argenx secures its second FDA approval for Vyvgart in autoimmune nerve disease. 🚨 Bristol Myers Squibb gains approval for Krazati, its KRAS drug for colorectal cancer. 🧪 iOnctura secures €80 million to advance treatment for rare eye cancer. 🧬 UroGen Pharma plans to raise $107 million for a new obesity biotech initiative. 👀 Sanofi and Belharra Therapeutics embark on a $700 million collaboration focusing on immunological diseases. #biotech #pharma #biopharma #genetherapy

🚨 🛎 Ups, downs, fatalities and breakthroughs... Another week in the biotech ecosystem. Here are some of the latest headlines ⬇ ▶ FDA advises doctors to discontinue us of Getinge life support pumps following more than 10 recalls and thousands or reported issues ▶ Novo Nordisk and Metaphore Biotechnologies collaboration worth $600M for 2 obesity drug treatments ▶ Disaster for MacroGenics, Inc. as phase 2 ADC trial leads to 5 deaths ▶ 3 year old boy suffers cardiac arrest death in Pfizer DMD gene therapy trial ▶ Gene therapy treatment to restore hearing shows promising signs following initial case studies produced by Regeneron ▶ Lykos Therapeutics PTSD treatment containing MDMA (otherwise known as the recreational drug Ecstasy) to be discussed by FDA next month ▶ Major breakthrough for Ring Therapeutics anellovectors which show signs of being re-doseable in primates What else is new? Comment below ⤵ ⤵ ⤵

GRI Bio Presents Positive Preclinical Data Demonstrating GRI-0621’s Ability to Inhibit Invariant Natural Killer T (iNKT) Cell Activity and Reduce Important Inflammatory and Fibrotic Drivers in Idiopathic Pulmonary Fibrosis. https://bit.ly/3zYseGw #NKT #IPF #FibroticDisease