#48 - CRISPR twins, criminal grins & clinical crises
The coffee break biotech roundup, by SomX.
All rise, court is now in session…
This week’s docket is crackling with controversy: calls to pause heritable genome editing have resurfaced, a former biotech board member faces insider trading charges, and Rocket’s gene therapy trial is halted after a tragic adverse event. But it’s not all grim, a sodium channel painkiller breaks through decades of dead ends, and a female-founded Parisian startup re-engineers glycans to outsmart immune rejection.
All adjourned (for now),
Dodo
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Discover 🔍
🧬 Gene editing leaders call for 10-year suspension of heritable human genome editing (FierceBiotech): Calls to pause heritable genome editing are back on the table, with leading voices from ISCT, ASGCT, and ARM. The move follows ongoing fallout from the CRISPR baby experiment in 2018, resulting in the birth of twin girls, citing unproven medical need, dodgy safety profiles, and the ethical minefield of editing entire family trees, not to mention the spectre of "designer babies" for the wealthy.
Our take: The call draws a critical distinction between therapeutic gene editing and permanent germline changes. Somatic editing is already transforming lives through breakthroughs in sickle cell and cancer treatments, but heritable editing ventures into dicey territory where mistakes can echo through generations. With therapeutic gene editing finally gaining serious momentum, premature germline ventures could trigger regulatory panic that drags down the whole sector.
⛓️ Former Chinook board member indicted for insider trading related to $3.2B Novartis buy (PharmaLive): Ross Haghighat has been hit with 19 criminal charges, including fraud and conspiracy, after allegedly trading on confidential information ahead of Chinook’s acquisition by Novartis. Prosecutors say he tipped off associates, raking in over $600,000 in the process. At the time, Haghighat sat on multiple boards, including Sernova, where he has since stepped down. The charges include conspiracy, fraud, and insider trading.
Our take: Biotech's cozy circles have struck again, but this time with federal charges. Haghighat juggled multiple positions while allegedly leaking intel through his network, and while that proximity is great to speed up deals, it also leaves room for slip-ups. And when sensitive terms go walkabout before the ink dries, the fallout is more than legal; reputations can be irreparably damaged. Trust can unravel quickly when governance can’t keep pace, and in this industry, access is everything.
🚀 Rocket’s Danon disease gene therapy on hold after patient death (BioSpace): Rocket has voluntarily suspended dosing in its RP-A501 trial after a patient developed fatal capillary leak syndrome, where fluid leaks from blood vessels causing dangerous drops in blood pressure. The FDA also placed a formal hold on the trail while the complication is investigated. Rocket is focusing on a novel immune suppressor recently added to the pre-treatment regimen that was designed to suppress the complement system before gene therapy induction.
Our take: Danon disease is a rare X-linked disorder with no approved treatments and a typical survival age <25. That context makes early safety trials especially sensitive, with risk and benefit closely scrutinised. This incident highlights how gene therapy is still in its adolescence, where scientific ambition and patient protection are still finding their balance. Optimistically, there is a strong case for continuing the research; however, more unfortunate missteps like these could lead investors to reconsider the entire field.
❤️🩹 The radical development of an entirely new painkiller (The New Yorker): Suzetrigine is the first drug approved to target NaV1.8, a sodium channel found in peripheral nerves. Where most painkillers dull the brain’s response or cast a wide systemic net, this one intercepts the signal before it ever reaches the central nervous system. Vitally, that sidestep steers clear of the brain’s reward circuitry, dodging the addictive traps that make opioids so risky and offering a long-awaited alternative for chronic pain patients.
Our take: NaV channels have tempted drug developers for over a decade. However, selectivity proved difficult, cardiac safety was a minefield, and trials rarely gave clear answers. Most efforts were shelved. Suzetrigine’s approval suggests that persistence – plus better screening tools and smarter trial design – can pay off. Ion channels may be back on the table, not just for pain, but for trickier conditions in psych, cardio, and metabolic diseases, too.
And finally…
🥐 Female founder in Paris secures €5.5M to advance drug safety with breakthrough biotech platform (TFN): Kyron.bio has secured seed funding from HCVC, Verve Ventures, Saras Capital, alongside a European Innovation Council award. The startup is tackling immunogenicity, one of the leading reasons biologics fail, by engineering glycan patterns on therapeutic proteins. Its platform delivers 97% uniformity in surface glycans, helping therapies evade immune surveillance and perform more reliably, all without interfering with standard manufacturing.
Our take: Patient immune systems routinely sabotage life-saving biologics, which is particularly troublesome for conditions requiring repeated dosing. Kyron sidesteps developing new drugs entirely, by creating a platform that makes existing therapies more durable. Precision glycosylation has previously been dismissed as too unpredictable for competitive advantage, but cracking this could reshape drug design and protect therapeutic assets in an increasingly crowded antibody market.
Tune in 🎧
🤝 Why Boehringer and GSK are betting on Ochre: Quin Wills shares why Ochre Bio’s human liver platform is attracting Boehringer and GSK and how RNA, AI and real organs are changing the future of liver disease drug development.
🖨️ 3D printing human organs and early detection of ovarian cancer: Vital3D’s Vidmantas Šakalys explains how their bioprinting tech moves from skin to organs, with cancer drug testing and tumour modelling already showing real-world impact.
🇪🇺 How to Ensure the Biopharmaceutical Sector Thrives in the EU: This episode explores 3D-printed organs with Vital3D’s Vidmantas Šakalys and how AOA Dx’s Oriana Papin-Zoghbi is chasing the first diagnostic test for ovarian cancer.
Apply ✍️
🧪 In Silico Principal Research Scientist, Immunocore: Fancy teaching machines the language of TCRs? Combine ML and structural biology to design smarter therapeutics, working across teams to transform rich datasets into real drug design breakthroughs.
🎓 Senior Lecturer/Reader, Manchester Institute of Biotechnology: Got group leader energy? Set your own research agenda, grow a team, and drive engineering biology forward with world-class facilities and support in one of the UK’s flagship biotech institutes.
👨💻 Bioinformatics Multi-omics Technical Lead, Illumina: Like finding signal in the biological noise? Lead the charge on multiomic analysis, building novel tools and algorithms that power Illumina’s cutting-edge assay development and unlock new insights from complex data.
RSVP 📆
🇬🇧 2-7.06 | SxSW | London, UK: Six days of science and tech innovation, creative brilliance, and networking opportunities – featuring talks, performances, screenings and immersive experiences. And if you haven’t got your ticket yet, use “SOMX50” to get 50% off all pass types!
🇨🇭 10-12.06 | Where Antibody and Protein Innovation is Driven to Commercial Success | Basel, Switzerland: Join Europe’s leading antibody event to discuss discoveries, engineering advancements and therapeutic developments for diseases such as neurodegeneration, metabolic disorders and autoimmune conditions.
🇺🇲 16-19.06 | The BIO International Convention | Boston, USA: The convention is the largest and most comprehensive event, bringing together 20,000 leaders representing the entirety of the global biotech industry.
🇩🇪 16-18.06 | 5th BIOTECH Conference 2025 | Freiburg, Germany: This year the conference will focus on bioprocess development and manufacture with single-use technology for biotherapeutics, cell and gene therapeutics and cellular agricultural products.
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