#46 - Gene genie
The coffee break biotech roundup, by SomX.
Hello from the lab that fell to Earth,
This week, it’s all about selective plays and smart positioning. GSK snaps up a liver asset just as the MASH market heats up, Stylus makes its gene editing debut with heavyweight backers, women’s health faces mixed signals despite funding gains, the London Cancer Hub starts taking shape, researchers test prenatal genetic therapy for SMA in mice, and a custom CRISPR treatment was delivered to a 6-month old in record time!
This is ground control signing off,
Dodo Stardust
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Discover 🔍
🧑🏻⚕️ GSK buys Phase III–ready liver drug in potential $2B deal to play in MASH arena (BioSpace): GSK is splashing the cash with a hefty upfront payment plus $800 million in success-based milestones for Boston Pharmaceuticals’ efimosfermin alfa, a once-monthly subcutaneous injection that mimics the metabolic hormone FGF21. The clever little drug regulates liver metabolic processes, reducing fat levels while calming inflammation and reversing fibrosis in MASH patients – and early data suggests it's rather good at it!
Our take: After years of false starts, FGF21 drugs are finally proving their worth. Despite earlier FGF21 analogs hitting immunogenicity hurdles, GSK’s timing couldn't be better, with Akero's recent cirrhosis reversal data and Madrigal's Rezdiffra claiming the first-to-market MASH crown. By nabbing this partially de-risked, late-stage asset, GSK neatly sidesteps those pesky early trial uncertainties and fast-tracks its path to market.
🧬 J&J, Eli Lilly join $85M financing for newly unveiled gene editing biotech Stylus (Fierce Biotech): Cambridge's Stylus Medicine is bursting onto the scene, combining a $40M series A and a fresh $45M extension with some ambitious plans for next-gen in vivo genetic medicines. Their platform combines therapeutic-grade recombinases, tailored payloads, and targeted LNP delivery. What’s more, they are set to showcase some preclinical data from a single dose of their CD3-targeted LNP cocktail containing both recombinase and CAR at ASGCT this week!
Our take: While the gene editing world has been utterly CRISPR-obsessed for a decade, Stylus is championing a refreshingly different approach. Their recombinase technology can neatly slot large DNA payloads into genomes with surgical precision, no tricky double-strand breakage required! For companies wrestling with complex genetic contexts, this could be the game-changer that finally delivers on safety, durability.
💸 Women’s health faces growing headwinds, despite jump in venture investment (BioPharma Dive): Conditions like endometriosis and PCOS remain frustratingly misunderstood – a telling symptom of how research for women-specific issues have been sidelined. Despite these persistent gaps, there's a glimmer of hope with record-breaking venture funding flowing into women's health startups in 2024! These promising funding upticks could finally support early science, but looming political challenges threaten this progress.
Our take: Diverse research creates better science. The historical exclusion of women from clinical trials means incomplete drug profiles and treatments that often miss the mark for half the population. With early-stage research now threatened by funding cuts and political meddling, we risk undoing the modest progress we've made. Several researchers have warned that dismantling the pipeline could take decades to fix.
💂 Plans for London Cancer Hub promises new destination for biotech innovation (Citeline): A rather impressive £1B development is creating a cosy neighbourhood where biotech companies can snuggle up next to the Institute of Cancer Research (ICR). This proximity gives biotechs access to patient data, clinical collaborations, lab equipment, and trial opportunities through The Royal Marsden. The ICR is eagerly hunting partners of all sizes whose scientists will be developing novel cancer-targeting inhibitors alongside their teams.
Our take: Too often, biotechs work in silos – cut off from clinicians and patients they're trying to help! The Hub's three-way marriage of The Royal Marsden, ICR, and commercial facilities offers direct access to clinicians, data, and trial infrastructure. This collaboration could make all the difference for commercial success. While most campuses lean toward either academia, business, or clinical care, the Hub's is balancing all three.
🐁 Prenatal genetic therapy for SMA shows promise in a mouse study (STAT): UCSF and Johns Hopkins have pulled off something rather clever – injecting an antisense oligonucleotide into pregnant mice with spinal muscular atrophy (SMA). The treatment travelled to the developing brains and spinal cords of mouse foetuses, improving motor function in the resulting pups. Unlike current treatments that battle the disease after birth, this approach tackles SMA in utero, addressing evidence that motor neurone degeneration begins before birth.
Our take: Delaying treatment until after birth means trying to repair damage that's already done. This new approach creates a system where scientists detect the genetic problem through prenatal testing, then fix it before symptoms occur. Beyond SMA, this could open the window for treating other neurodegenerative conditions, as prenatal genome sequencing becomes commonplace – potentially transforming unfortunate genetic forecasts into treatable conditions.
And finally…
👶🏻 A baby received a custom CRISPR treatment in record time (WIRED): Last August, baby Muldoon was born with severe CPS1 deficiency – a condition causing ammonia buildup in the blood. Six months later, the little chap received a personalised CRISPR treatment! Current treatment options for the disease, which include restrictive diets and liver transplants, aren't exactly ideal. So, after safety testing, the team rushed to the FDA. They applied on Valentine's Day, were approved on February 21, and administered on February 25 – now that's a medical express lane!
Our take: It usually takes years to design, test, and approve a gene therapy. Baby Muldoon’s treatment went from concept to clinic in just 6 months! That speed shows how far in vivo editing has come over the years – all thanks to advanced sequencing, base editors, and liver-homing lipid nanoparticles. For ultra-rare disorders, this incredible story shows that fast timelines, feasibility, and access are possible. Working on ways to reduce costs should be next on the agenda.
Tune in 🎧
🗞️ STAT News, the dynamics of biotech journalism and how it compares to other industries: What makes biotech journalism tick? A look at investor-driven storytelling, editorial independence, and how writing about science differs from covering Big Tech.
💉 The DSCSA deadline and MoonLake's Nanobod: Delve into looming DSCSA compliance challenges and a pioneering Nanobody programme targeting inflammatory diseases, as experts navigate regulatory hurdles and blockbuster potential.
👁️ Why Sam Altman Wants to Scan Your Eyeball: AI is evolving fast – too fast to tell bots from people. Could biometric ID be the fix, or just fuel more privacy panic?
Apply ✍️
👑 Appointment of Board Chair, The Rosalind Franklin Institute: Enjoy connecting scientific dots at the highest level? Guide a health research institute's strategic vision while building key relationships across government, industry, and academia.
🗂️ Policy Team Coordinator, BioIndustry Association: Love being the glue that holds everything together? Coordinate vital policy committees, manage member communities, and keep the team running smoothly as they champion UK's biotech landscape.
📈 Biostatistics Senior Manager, Amgen: Good at storytelling with statistics? Develop analytical capabilities, advise on trial design, and collaborate across teams to help innovative medicines reach patients.
RSVP 📆
🇬🇧 21-22.05 | CHEM UK 2025 | Birmingham, UK: An opportunity to engage with 550 exhibitors and 100 leading experts in the chemicals, process engineering, and formulated product industries.
🇩🇪 22-23.05 | Global Bioprocessing & Bio Technology Summit | Berlin, Germany: This leading event will unite leading industry experts, researchers, and innovators to explore the latest breakthroughs in bio manufacturing.
🧭 2-7.05 | 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!
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We’re SomX – a communications and creative agency trusted by biotech, pharma and healthtech pioneers. We craft strategy, content, PR and design that translate complex biology into compelling stories and investor‑ready excitement. Get in touch to amplify your science beyond the lab bench.