Fabian Krebs

Roche Drops Early Obesity Candidate Over Competitiveness Concerns❗ Roche has announced it will discontinue development of CT-173, a PYY mimetic for obesity, citing that it didn’t meet the company’s bar for developability and competitiveness. CT-173 was acquired in Roche’s $2.7B Carmot Therapeutics, Inc. deal in 2023. Despite promising early data combining CT-173 with its lead GLP-1/GIP dual agonist CT-388, Roche decided to cut the asset to sharpen focus on more promising obesity programs. Teresa Harris Graham, CEO of Roche, emphasized this decision has “very little impact on the overall obesity portfolio” — which still includes CT-388, expected to deliver Phase III-enabling data by year-end.

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👨‍🔬💬 𝐂𝐞𝐥𝐥 & 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐏𝐨𝐬𝐢𝐭𝐢𝐯𝐢𝐭𝐲: 𝟐𝟐𝐧𝐝 𝐉𝐮𝐥𝐲 𝟐𝟎𝟐𝟓 👩‍🔬💬 🧬 AAVantgarde received FDA clearance to begin a Phase 1/2 clinical trial for AAVB-039, its gene therapy candidate for Stargardt disease, a common inherited form of macular degeneration. The therapy uses AAVantgarde’s dual AAV intein platform to deliver the large ABCA4 gene, aiming to address the genetic root cause of the condition. This is their second program to enter the clinic, alongside AAVB-081 for Usher syndrome type 1B. 🧬 Genascence Corporation received RMAT designation for GNSC-001, its gene therapy candidate for knee osteoarthritis (OA). GNSC-001 uses an AAV vector to express IL-1Ra, a protein that blocks IL-1, a key driver of inflammation and cartilage damage in OA. Designed for long-term efficacy via a single joint injection, GNSC-001 has shown sustained therapeutic IL-1Ra levels in early trials. 🧬 Rocket Pharmaceuticals received RMAT designation for RP-A601, its investigational gene therapy for PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM), a life-threatening genetic heart disease. RP-A601 uses an AAVrh74 vector to deliver a functional PKP2 gene via a one-time IV infusion. Early Phase 1 data show strong safety, increased PKP2 expression, improved heart structure, and better clinical outcomes. 🦠 CytoMed Therapeutics Limited successfully completed dose level 1 of its first-in-human Phase I ANGELICA trial for CTM-N2D, an allogeneic CAR gamma delta T cell therapy targeting NKG2DL, and will advance to dose level 2 in Q3 2025. CTM-N2D is derived from healthy donors and modified to target cancer-associated antigens found in both solid tumors and blood cancers. Unlike conventional autologous CAR-T therapies, CTM-N2D does not require patient-donor matching. 🧬 NKGen Biotech, Inc. received FDA authorization for an Expanded Access Program (EAP) allowing the use of its investigational autologous NK cell therapy, troculeucel, in up to 20 patients with various neurodegenerative diseases. While troculeucel is currently in a Phase 2a trial for moderate-stage Alzheimer’s disease, the EAP extends access to earlier-stage Alzheimer’s and other conditions including Parkinson’s, ALS, MSA, PSP, FTD, CBD, MS, and LBD. 🧬 Sanofi’s gene therapy candidate SAR446597 has received FDA Fast Track designation for geographic atrophy caused by age-related macular degeneration (AMD). SAR446597 is a one-time intravitreal gene therapy delivering antibody fragments that inhibit two key components of the complement pathway (C1s and factor Bb) aiming to reduce inflammation and retinal damage. This dual-targeting strategy may offer sustained therapeutic effects and reduce the need for repeated injections. 🛎 My aim is to be the messenger when it comes to your CGT news. Follow #mCGT to stay updated! #celltherapy #genetherapy #ATMP #advancedtherapy

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🚨🧠 Neuroscience News – 7 day recap: 08/08 ––– 𝘪𝘯 𝘱𝘢𝘳𝘵𝘯𝘦𝘳𝘴𝘩𝘪𝘱 𝘸𝘪𝘵𝘩 World CNS Series In typical post-conference fashion, not a whole lot of news this week so no newsletter, but plenty to cast your eyes over. See below, including the unfortunate Vertex Phase 2 news 🥼 Clinical News Vertex Pharmaceuticals read out topline data of their NaV1.8 inhibitor, VX-993, from their Phase 2 trial targeting acute pain with data showing it missed its primary endpoint, failing to show a statistically significant improvement in the Sum of the Pain Intensity Difference scale after 48 hours of treatment BioXcel Therapeutics, Inc. finished their Phase 3 trial evaluating BXCL501, targeting selective alpha-2 adrenergic receptors, targeting agitation associated with bipolar or schizophrenia. Topline data expected this month with expectations to support their sNDA Praxis Precision Medicines, Inc. reported Phase 2 topline data evaluating vormatrigine target focal epilepsy. Data showed vormatrigine reduced seizures by 56.3% and roughly 22% of patients seizures completed stopped over the last 28 days of treatment. Vormatrigine is a small molecule targeting the hyperexcitable state of sodium-channels Click Therapeutics, Inc. and Boehringer Ingelheim prescription digital therapy, CT-155, met primary endpoint in a Phase 3 trial designed to address the negative symptoms of schizophrenia Dyne Therapeutics have been granted Breakthrough Therapy Designation from the FDA for DYNE-251 targeting DMD Teva Pharmaceuticals announced the FDA has approved AJOVY targeting episodic migraine in children and adolescent patients aged 6-17 years old who weight 45KG or more Cingulate Inc. submitted their NDA to the FDA for CTx-1301 targeting ADHD 💰 Financial News Lysoway Therapeutics have been granted $2.93M from The Michael J. Fox Foundation for Parkinson's Research to target TRPML1. FIH expected Q1 2026 Sanofi completed the well documented acquisition of Vigil Neuroscience 🤝 Collaborations Pendopharm signed a distribution agreement with Neuraxpharm for the commercialization of Buccolam – indicated for the treatment of prolonged, acute, convulsive seizures in children 

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It's Friday, which means it's time to get back to our 🧬𝗦𝘁𝗮𝗿𝘁-𝘂𝗽 𝗼𝗳 𝘁𝗵𝗲 𝗪𝗲𝗲𝗸 series! This week it’s Orbis Medicines, a company I’ve been following for a few years as I’ve also helped grow their Swiss team. (Btw, this is what I find most exciting about working with startups: you get to see their trajectory over just a few years, watch how their strategy evolves and adapts to the market, celebrate their milestones, and understand the challenges they face along the way). 👉 But let's go back to Orbis: they're tackling a tough problem in drug discovery. 𝗠𝗮𝗰𝗿𝗼𝗰𝘆𝗰𝗹𝗲𝘀  (cyclic compounds with incredible binding versatility) have long been 𝗵𝗮𝗿𝗱 𝘁𝗼 𝘁𝘂𝗿𝗻 𝗶𝗻𝘁𝗼 𝗼𝗿𝗮𝗹𝗹𝘆 𝗮𝘃𝗮𝗶𝗹𝗮𝗯𝗹𝗲 𝗱𝗿𝘂𝗴𝘀 that can reach intracellular targets (and also reach more patients!). Their answer is 𝗻-𝗖𝘆𝗰𝗹𝗲𝘀, molecules that sit 𝗯𝗲𝘁𝘄𝗲𝗲𝗻 𝘀𝗺𝗮𝗹𝗹 𝗺𝗼𝗹𝗲𝗰𝘂𝗹𝗲𝘀 𝗮𝗻𝗱 𝘁𝗿𝗮𝗱𝗶𝘁𝗶𝗼𝗻𝗮𝗹 𝗺𝗮𝗰𝗿𝗼𝗰𝘆𝗰𝗹𝗲𝘀, with better cell permeability and oral bioavailability. Their n-Gen platform lets them design, make, and test these compounds quickly, using AI and machine learning to navigate the near-infinite chemical space efficiently. 🤒💊 Their focus? Their macrocycle candidates will go for targets already validated by blockbuster biologics in 𝗜𝗺𝗺𝘂𝗻𝗼𝗹𝗼𝗴𝘆 & 𝗜𝗻𝗳𝗹𝗮𝗺𝗺𝗮𝘁𝗶𝗼𝗻 𝗮𝗻𝗱 𝗖𝗮𝗿𝗱𝗶𝗼𝗺𝗲𝘁𝗮𝗯𝗼𝗹𝗶𝗰 disease. On top of that, they’re exploring a wider range of therapeutic areas, tackling both intra- and extracellular targets with high potential. Thus far Orbis has had an impressive journey: they recently closed a €𝟵𝟬𝗠 𝗦𝗲𝗿𝗶𝗲𝘀 𝗔 and, from Lausanne (Switzerland) have 𝗲𝘅𝗽𝗮𝗻𝗱𝗲𝗱 𝘁𝗼 𝗗𝗲𝗻𝗺𝗮𝗿𝗸, 𝗰𝗼𝗹𝗹𝗮𝗯𝗼𝗿𝗮𝘁𝗶𝗻𝗴 𝘄𝗶𝘁𝗵 𝗔𝗜 𝘀𝘂𝗽𝗲𝗿𝗰𝗼𝗺𝗽𝘂𝘁𝗶𝗻𝗴 𝗿𝗲𝘀𝗼𝘂𝗿𝗰𝗲𝘀 and building a strong team. Definitely one to watch! 👀 ________________________________ 🧬 𝗛𝗶, 𝗜’𝗺 𝗖𝗲𝗰𝗶𝗹𝗶𝗮 and help scientists transition into biotech. Follow me for industry insights and tips on moving into this space! If you need support or want to collaborate, I’m always happy to have a chat. Morten Graugaard Sevan Habeshian Daniela Brodbeck Chiara Toniolo Sara Cristinelli João Ribas Vanessa Carle #Biotech #DrugDiscovery #OralTherapeutics #Macrocycles #PrecisionMedicine #NextGenTherapeutics #IntracellularTargets #LifeSciences #StartupOfTheWeek

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🧠🚨 CNS News 🚨🧠 AbbVie has just made another big move in neuroscience, agreeing to acquire Gilgamesh Pharmaceuticals bretisilocin program in a deal worth up to $1.2 billion. Rather than a full acquisition, AbbVie will take over bretisilocin (GM-2505), while Gilgamesh spins out its other assets into a new entity. Bretisilocin recently hit a 94% remission rate in a Phase 2 trial for major depressive disorder, with patients dosed just twice, two weeks apart. It acts on the 5-HT2A receptor like LSD/psilocybin, but with a shorter half-life, potentially making it more practical for in-clinic use, similar to the model J&J established with Spravato. Could shorter acting psychedelic therapies like bretisilocin be the breakthrough psychiatry has been waiting for? #Psychedelics #CNS #Neuroscience #Biotechnology

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🚨 Big News in Hypertension Treatment 🚨 AstraZeneca is gearing up for an FDA filing after releasing late-stage results on baxdrostat, its new hypertension pill and the data is impressive: ✅ Nearly 10 mmHg reduction in blood pressure at the higher dose ✅ Consistent results across uncontrolled & treatment-resistant groups ✅ Almost 3x the odds of patients hitting their target SBP (<130 mmHg) ✅ Potential beyond hypertension: being evaluated for CKD & heart failure prevention This comes just two years after AstraZeneca acquired CinCor Pharma, Inc. for $1.3B this is a bold move that now looks like it’s paying off. If approved, baxdrostat could become a game-changer in cardiovascular care. 🔔 I post weekly Life Science news in the Manufacturing and Engineering space. Follow me to stay up to date! #LifeSciences #Pharma #AstraZeneca #Hypertension #ClinicalTrials #DrugDevelopment #FDA #HealthcareInnovation

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The journey of bringing a Medical Device to market Long, uncertain, and never easy 1️⃣ Spotting a problem worth solving: Can it be built, and does it matter? Is there unmet demand in the market? How big is the market? 💰 Grants, Friends & Family, Pre-Seed 2️⃣ Turn the concept (idea) into a working product. Validating & proving the technology can uphold. 💰 Seed 3️⃣ Regulatory/Clinical One of the hardest sections- getting it wrong can cost $$$$$$s. Building Quality systems, designing clinical trials, and navigating country approvals. Typically multiple raises to expand clinical studies, manufacturing scale-up and infrastructure for commercialisation. 💰 Series A/B+ 4️⃣ Commercialization Launching into the market, scaling operations and meeting sales demand. Designing & Executing your Market Access strategy. 💰 Growth Capital / Series C / Exit (IPO) + the pressures of meeting investor timelines, ensuring compliance, and keeping enough of a runway (& more!) Over the last 5 years, 74 U.S. Medical Device companies achieved IPO But research shows that 75% of medical device companies fail or never make it ot market In most cases, founders have a brilliant vision but are unable to assemble the right team of professionals to build, test, and market their devices We specialize in building your founding team- making that idea, a reality. And putting you in the top 25% And when you reach IPO, the hard work & climb was worth it! Danielle Lea

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🚀 New Partnership Alert Arkivum has teamed up with PHARMASEAL to deliver a complete end-to-end clinical trial technology solution. By combining Arkivum’s GxP-validated digital archiving with PHARMASEAL’s Engility CTMS & eTMF, sponsors can now: ✅ Build data-retention planning in from day one ✅ Ensure long-term integrity, accessibility & inspection readiness ✅ Stay fully compliant with ICH E6 (R3), EU CTR, and evolving regulations “This partnership will increase awareness of the importance of a fit-for-purpose solution to preserve all essential clinical trial records and data,” said Chris Sigley, CEO of Arkivum. Ricky Lakhani, Chief Product Officer at PHARMASEAL, added, “It guarantees that critical trial records remain accessible, compliant, and usable for decades to come.” This collaboration future-proofs clinical trial records, keeping essential data secure and usable for decades. #ClinicalTrials #LifeScienceNews #BioPharma #CRO

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