#43 - Brainy breakthroughs and schizophrenia stumbles
The coffee break biotech roundup, by SomX
Good morning you brainy lot!
This week, we bring news of the first use of CRISPR therapy in the human brain(!), alongside billion-dollar investments, significant breakthroughs in oncology and leukaemia treatment, and disappointing clinical trial results for Bristol Myers Squibb's hotly anticipated schizophrenia treatment.
Until the next cognitive leap,
Dodo
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Discover 🔍
🇨🇳 China Startup Injects CRISPR Therapy into Human Brain for the First Time (Endpoints): Shanghai’s little-known HuidaGene Therapeutics has made history; using a CRISPR-based therapy in the brain – for the very first time – to treat a 9-year-old with MECP2 duplication syndrome, a rare and deadly neurodevelopmental disorder. Within 12 weeks of treatment, the child showed improved motor skills and responsiveness, with no serious adverse events reported. The therapy balances gene expression by targeting short-lived mRNA molecules of the MECP2 gene, to ensure it is neither over- or under- expressed.
Our take: This incredible neurological feat marks the world’s first human use of CRISPR in the brain, and only the second time RNA-targeting CRISPR (Cas13Y) has been used in people. But HuidaGene is not stopping there: with a deep toolkit of CRISPR enzymes for DNA editing and RNA knockdown, and a suite of gene editing tools at their disposal – as well as impressive human proof-of-concept data – they’re now looking to partner with companies that could help them advance to Phase 3 trials. We, for one, will be eagerly watching their next steps!
🤑 Swiss biotech Veraxa vaults to Nasdaq listing with SPAC deal (pharmaphorum): Veraxa Biotech is set to go public on Nasdaq thanks to a $1.64 billion SPAC merger with Voyager, in a move that will unlock $253 million in funding to advance its next-gen cancer therapies. The company’s nifty BiTAC platform designs therapies with two complementary antibody precursors. The drug molecule only become therapeutically active when both antibody targets are present on nearby cancer cells, acting as a built-in safety switch to minimise side effects.
Our take: Veraxa aims to address one of immuno-oncology’s most stubborn challenges: balancing efficacy with safety. Their double target antibody approach could help sidestep the off-target effects that plague bispecifics and ADCs, offering a promising path to higher dosing and broader patient populations in the immuno-oncology space. Very smart!
🦀 Boehringer inks Tessellate deal to slot synthetic lethal program into oncology pipeline (Fierce Biotech): Boehringer Ingelheim has struck a $570 million deal with Tessellate Bio to develop cutting-edge cancer therapies, with a focus on tumours driven by alternative lengthening of telomeres (ALT), which affects 10–15% of cancers. Tessellate’s small-molecule inhibitors aim to selectively destroy ALT-dependent cancer cells while sparing healthy tissue, offering hope for patients with hard-to-treat tumours.
Our take: Boehringer’s partnership with Tessellate Bio signals both the maturation of synthetic lethality as a therapeutic strategy and the growing appetite for targeting previously “undruggable” cancer vulnerabilities. By focusing on ALT, a largely untapped biological pathway, the alliance could lead to breakthrough precision treatments for cancers that have limited treatment options, especially those resistant to current therapies like PARP inhibitors.
🧬 Novel Leukaemia Target Found in RNA-Binding Cell Surface Protein (GEN): Scientists have uncovered a game-changing cancer target: a normally hidden RNA-binding protein, NPM1, that appears in abundance on the surface of leukaemia and some solid tumour cells. By using monoclonal antibodies to home in on these exposed proteins, researchers wiped out leukaemia in bone marrow samples and mouse models, while sparing noncancerous cells. This breakthrough could open the door to safer, more precise treatments for hard-to-treat cancers like acute myeloid leukaemia, prostate, and colorectal cancer.
Our take: The discovery that cell-surface glycoRNAs can serve as potent, selective targets for cancer therapy marks a quantum leap forward in the quest for safer, more precise immuno-oncology treatments. For years, scientists have sought elusive “molecular handles” that can be safely leveraged to unlock immune attacks on tumours without collateral damage. The identification of NPM1 may finally provide that key, while also unlocking valuable routes for detection and monitoring of disease.
And finally….
🧠 Bristol Myers’ prized schizophrenia drug stumbles in testing (BioPharma Dive): Bristol Myers Squibb’s high-profile schizophrenia drug Cobenfy – acquired just last year for $14 billion – has stumbled in a 400-person clinical trial, where it failed to outperform the placebo. The result, which showed only a modest two-point improvement on a standard symptom scale, prompted analysts to slash long-term sales forecasts, with some cutting 2030 estimates by over half.
Our take: Well, this clinical setback has sent BMS shares tumbling, and certainly dents their hopes for blockbuster therapetic expansion. But while the optics certainly aren’t bright, the pharma giant still sees potential for Cobenfy in a “myriad” of other indications, including bipolar disorder, autism spectrum disorder and Alzheimer’s psychosis, and hopes the results “may provide valuable direction in our ongoing search for complementary approaches to address persistent treatment gaps”. Time will tell if this is just a snag along the road, or a gamble that didn’t pay off.
Tune in 🎧
🧑🔬 Synbio Companies Come to Grips with the One “Omic” that Matters: The synthetic biology sector is facing headwinds, sparking concerns that the US could fall behind China. SynBioBeta's CEO breaks down the current state of the industry in this insightful podcast episode.
🌊 A Between the Biotech Waves Conversation with Kent Rogers: Newly minted CEO of EveryONE Medicines, Kent Rogers, shares insights from his 30+ year career, and discusses EveryONE’s mission to develop individualised precision therapeutics for children with rare diseases.
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🧑🔬 Biostatistician (PhD, Experienced) Medpace: Looking for a new role in biostatistics? With a focus on clinical trials, this role offers an opportunity to join a growing company accelerating the global development of safe and effective medical therapeutics.
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🇬🇧 28-30.04 | LSX World Congress 2025 | London, UK: LSX brings together global leaders in life sciences and biotech for networking, discussions and innovation showcases.
🇨🇭5-6.05 | Swiss Biotech Day 2025 | Basel, Switzerland: A large-scale networking event for life science professionals, featuring exhibitors, startups, and discussions.
🇧🇪 12-14.05 | Bio€quity Europe 2025 | Brugge, Belgium: The event is a key networking and partnering event for the biopharma industry, bringing together investors and company representatives.
🇩🇪 2-14.05 | CIMT Annual Meeting | Mainz, Germany: Europe's largest non-profit event focused on cancer immunotherapy R&D, this meeting gathers the global community for scientific exchange, collaboration and education.
🌍 22-23.05 | The Global Bioprocessing Summit 2025 | Berlin, Germany: Join top industry leaders and researchers to explore cutting-edge technologies and strategies that will drive efficiency and sustainability in biomanufacturing.
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