#35 - Risk scores, rare diseases, and ripe tech

The coffee break biotech roundup, by SomX.

Gooday, my fruitful readers!

This week’s stories are all about ripening – whether tech, investments, or actual bananas. Healx’s AI cancer drug reaches its first patient dose, Bupa offers the first UK genetic test for disease prediction, OrganOx secures funding for liver transplants, bluebird bio might be past its best, and gene-edited bananas are staying fresher for longer.

Time to split,

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🤖 History beckons as Healx doses first rare cancer patient with AI-created drug (Business Weekly): Cambridge-based Healx has dosed the first patient in INSPIRE-NF1, a Phase 2 trial for HLX-1502. This oral treatment targets neurofibromatosis type 1 – nerve tumour causing genetic disorder that affects 1 in 2,500 people worldwide. Founded by biochemical engineer Tim Guilliams and Viagra co-inventor David Brown, Healx has already secured FDA Fast Track, Orphan Drug, and Rare Pediatric Disease designations for this promising therapy.

Our take: Rare diseases rarely attract big pharma budgets, but Healx is challenging the status quo. NF1’s biology is notoriously complex, and conventional drug development has struggled to keep up. Healx is hoping that not only could their therapy help improve NF1 outcomes, but also that it could present a shift in rare disease drug discovery – turning it from a high-risk gamble into a data-driven opportunity for investment.

🧬 Bupa to offer first genetic test for disease prediction in UK (The Times): Polygenic risk scores are entering mainstream healthcare, with Bupa offering customers a genetic test that predicts disease risk for conditions like cardiovascular disease, diabetes, and cancer. Developed by Oxford spinout Genomics, led by Professor Sir Peter Donnelly (Wellcome Centre for Human Genetics), and member Professor Dame Sally Davies (former Chief Medical Officer for England), the test is pending UK registration but is already widely available in the US.

Our take: 35 years after the Human Genome Project began, genetic risk scoring is now entering the toolkit of disease prevention. Bupa is hoping that its insights will steer high-risk patients to earlier intervention and strengthen the UK’s preventative care programmes. What remains to be seen is if this will translate to better health outcomes for all, or further entrench disparities in insurance and healthcare access.

💰 OrganOx raises $142m to accelerate growth of metra platform technology (Medical Device Network): The Oxford University spinout just secured an oversubscribed financing round led by HealthQuest Capital, with a mix of new investors. Their metra platform – already used in 5,000+ liver transplants – keeps donor organs functioning outside the body, allowing pre-transplant evaluations. However, OrganOx isn't stopping there: they're kicking off a first-in-human clinical trial with biotech eGenesis, focusing on genetically engineered pig livers for acute-on-chronic liver failure.

Our take: Liver transplants are finally getting their much-needed upgrade. By keeping organs "warm," metabolically active, and directly assessable for up to several hours, OrganOx’s platform improves viability, reduces discard rates, and maximises the use of donor livers. The technology – which increases the number of organs available for transplant – is to be extended into kidneys too, with US clinical trials starting soon.

📉 Once valued at $10B, bluebird bio sold to private equity firms for $29M (Fierce Pharma): Oh, how the mighty have fallen. Once a poster child for gene therapy, bluebird bio has been scooped up for just $3.00 per share (falling from a $2,300 high) after failing to build a sustainable market for its three high-cost treatments. There is however a silver lining: if annual sales reach $600M by 2027, shareholders will nab an extra $6.84 per share.

Our take: Bluebird’s treatments broke scientific ground but never quite built the commercial foundation to justify their multimillion-dollar price tags. Having just been denied, for the third time, a priority review voucher (PRV) from the FDA for its sickle cell disease treatment Lyfgenia – and competing against Vertex’s cheaper alternative – bluebird’s private equity backers will need more than just sales incentives to turn this story around.

And finally…

🍌 Tropic to launch non-browning bananas in March, extended shelf-life bananas by year-end (AgFunder News): Plant biotech Tropic is launching gene-edited Cavendish bananas in a bid to cut waste. Using CRISPR, they company has managed to reduce browning and ripening by disabling the genes responsible for producing ethylene and polyphenol oxidase. Additionally, their GEiGS RNAi tech targets fungal disease TR4, which is decimating crops around the world. With regulatory approvals in place in the Philippines, Colombia, Honduras, the USA, and Canada, they’re now scaling up with suppliers.

Our take: Just as Panama disease wiped out the Gros Michel banana in the 1950s, the conventional Cavendish banana is now at risk due to the rise of TR4 disease. Thankfully, Tropic is ahead of the curve, and is working with some of the world’s biggest banana producers on their TR4 resistant bananas. If successful, Tropic’s model could change how gene editing is applied across global staple crops – reducing waste, extending viability, and improving food security.

Tune in

🔋 Solving AI’s Energy Problem

Neil deGrasse Tyson, Gary O’Reilly & Paul Mecurio discuss the physics, safety, and future of nuclear reactors in a world of increasing power demands, with nuclear engineer Kathryn Huff (free on Apple Podcasts).

🥊 The Showdown Between Elon Musk and Sam Altman
Lauren Goode, Michael Calore and Zoë Schiffer discuss the relationship between Sam Altman and Elon Musk and where it all started at OpenAI, then just a non-profit research lab.

🏦 Making Sense Of Federal Cuts To Science – And What Comes Next

Host Flora Lichtman and Dr. Sudip Parikh discuss what’s next for american science and tech, what cuts for “efficiency” are getting wrong, and what’s at stake in the next few weeks.

Apply ✍️

🦻 Scientist/Senior Scientist, CamGene Therapeutics: Got an ear for mRNA therapies? Design and test cutting-edge gene therapies to restore hearing, bridging biotech innovation with academic research.

💼 Global Strategic Account Specialist, Cytiva: Got a knack for high-stakes biotech deals? Lead strategic partnerships, negotiate complex IP agreements, and help bring cutting-edge therapies from research to market.

RSVP 📆

🇪🇺 10-11.03 | 35th Annual European Pharma Congress 2025 | Rome: A dynamic two-day gathering of global pharmaceutical leaders, researchers and innovators to shape the industry's future.

🏴󠁧󠁢󠁳󠁣󠁴󠁿 12-13.03 | IBioIC’s 11th Annual Conference | Glasgow: IBioIC's 11th Annual Conference unites 400+ bio-industry experts to discuss successful partnerships and bio-based product commercialisation.

🇮🇹 17-19.03 | BIO-Europe Spring | Milan: The conference promises a dynamic blend of cultural heritage and cutting-edge life sciences, offering 3,700+ industry leaders unparalleled networking opportunities through meetings, receptions, and wellness activities.

👩🏻‍💻 18.03 | FemHealth Integrates | Manchester: Tackling decades-long inequalities in healthcare, FemHealth Integrates is the perfect opportunity for people of all genders to come together to bring about greater equality in biotech and healthcare.

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