#65 – Rare diseases, rescued royalties, and revived relics
The coffee break biotech roundup, by SomX.
Hello, my resilient revivalists,
This week’s roundup pulses with new forms of survival: a £10M commitment gives childhood cancer treatments the spotlight they deserve, Bluebird bio is reborn as Genetix under private equity’s wing, Xoma finds profits in biotech shutdowns, a one-time gene therapy shows striking Huntington’s results, and Colossal edges closer to resurrecting my feathered kin.
Until the next chirp,
Dodo
If there’s anything you’d like to see in future editions of Biotech Dodo, send us a message.
Discover 🔍
👶🏻 C-Further announces major partnership with GOSH Charity to drive innovation in childhood cancer treatment (C-Further): GOSH Charity, the UK’s largest child health funder, has pledged £10M to strengthen a global consortium tackling paediatric cancers – an area often neglected by mainstream drug discovery. The commitment lifts C-Further’s total funding pot to £37M and will support a broad therapeutic portfolio, with first projects set to be revealed later this year, giving long-overdue focus to childhood cancer treatments.
Our take: Childhood cancer isn’t just “adult cancer, but smaller.” Most paediatric cancers are biologically worlds apart from their adult counterparts, yet kids often end up with hand-me-down therapies or decades-old protocols with brutal long-term effects. GOSH Charity’s involvement brings serious clinical infrastructure and patient communities into the fold, creating an ecosystem where early-stage discovery can actually talk to real-world clinical needs.
🐦 Bye bye Bluebird: Gene therapy biotech emerges from private buyout with rebrand (BioSpace): Bluebird bio has flown the coop – sort of. After a cash crunch led to a $50M private equity buyout by Carlyle and SK Capital Partners, the company has shed its feathers and returned to its 1992 roots as Genetix Biotherapeutics. The rebrand is focused on making its three approved gene therapies – Lyfgenia, Skysona, and Zynteglo – accessible, with plans to expand manufacturing capacity within the year.
Our take: This is private equity’s first real test at salvaging a commercial-stage gene therapy company rather than chopping it up for parts. Most struggling firms either collapse or get liquidated, but Carlyle’s betting they can fix the unglamorous stuff – cryopreservation logistics, scheduling efficiencies, and getting hospitals ready for $3M therapies. It’s a big shift from “does this vector work?” to “can healthcare systems actually handle it?”.
🧟 Why Xoma, a drug royalty firm, is hunting biotech ‘zombies’ (BioPharma Dive): The royalty firm has found a lucrative niche in biotech’s brutal fundraising environment: swooping up distressed companies and liquidating them for profit. Xoma acquired 6 biotechs in 2024 alone, including Kinnate Biopharma and Pulmokine, making money by winding down operations and selling off leftover IP. With nearly 300 biotechs now trading below their cash reserves, frustrated investors are increasingly pressuring management to accept buyouts.
Our take: What used to be an embarrassing drift into irrelevance has become a structured exit strategy, and Xoma’s turned corporate euthanasia into an art form. Boards are finally accepting that sometimes the kindest thing is dignified liquidation rather than years of shuffling. And by reshaping failure from drawn-out agony into efficient capital recycling, more money gets freed up for the next wave of biotech hopefuls.
🧬 Gene therapy appears to slow Huntington’s disease progression (UCL News): MT-130 has delivered some genuinely jaw-dropping results in a Phase I/II trial, with treated Huntington’s patients showing 75% less disease progression compared to matched controls over three years. Even better, neuronal damage markers actually decreased in treated folks, bucking the usual 20-30% increase. The one-time gene therapy, delivered directly to the brain via surgery, was well-tolerated across 29 patients. FDA accelerated approval application lands early next year.
Our take: The surgical brain delivery model is something that looked bonkers a decade ago but now seems downright sensible for monogenic neurodegeneration. The one-time AAV treatment creates a blueprint that could work for spinocerebellar ataxias, frontotemporal dementia subtypes, and other “impossible” targets. It seems gene therapy is graduating into properly serious neurological territory.
And finally…
🦤 Resurrection of dodo bird now one step closer, claims Colossal Biosciences (Phys.org): In a breakthrough for my feathery ancestors, Colossal has been successful in growing primordial germ cells from rock doves for a full 60 days after screening over 300 culture “recipes”. They’ll gene-edit germ cells from Nicobar pigeons (my closest living relative) to reconstruct dodo-like genomes, then inject these into chicken embryos as feathered surrogates. Eventually, they aim to rewild these “neo-dodos” on Mauritius, though naysayers say they’ll just be glorified GMO pigeons.
Our take: Hooray! This feels like great progress from de-extinction experts Colossal (who we first covered back in issue 1). Mastering avian germline engineering in non-model species means those same protocols could rescue our endangered cousins – like the pink pigeon, whose gene pool needs serious help – and also improve avian vaccine production and agricultural breeding. Speaking for all Dodo’s, it will be a delight to have company back on Mauritius!
Tune in 🎧
⚡️ Faster Science, Better Drugs: Clinical trials are still too slow, too costly, and too prone to failure. This episode digs into patient registries, data infrastructure, and smarter regulation as potential lifelines.
💉 100 years of insulin in 15 minutes: From hams to hormones, this episode traces insulin’s journey from pig pancreases to engineered bacteria. Listen to a century of biomanufacturing, breakthroughs, setbacks and the medicine that changed diabetes forever.
🩺 Breaking Down Achondroplasia: Paediatric geneticist Dr. Janet Legare covers how the condition affects bone growth, current treatment options, the role of FGFR3 mutations, and the importance of early diagnosis and support.
Apply ✍️
🧬 Director, Clinical Scientist - Oncology, GSK: Know your way around cells and study shells? Lead global oncology studies, shape strategy from protocol to publication, and guide teams translating immune insights into patient outcomes.
🥜 Allergy Scientist, Siemens Healthineers: Allergic to boring jobs? Master the art of allergen extraction and purification, turning proteins into powerful diagnostic tools while enjoying top-tier benefits and the satisfaction of helping people breathe easier.
🤖 Senior Product Manager – Applied AI, Owkin: Ready to be AI’s biology tutor? Shape cutting-edge agentic copilots that help researchers discover life-saving treatments, working with a $300M-backed team across Paris, London, or remotely.
RSVP 📆
📈 30.09 | Sidley Healthcare Investment Conference 2025 | London, UK: Join Sidley’s one‑day London event uniting global pharma, biotech, healthcare and investors to explore the deals, trends and innovations shaping life sciences success.
👩🏻🔬 01.10 | Women in Biotech | Oxford, UK: Although investment in women’s health is rising, a funding gap persists. Meet founders at the Natural History Museum for timely debate and networking; all welcome.
🧪 21-22.10 | Drug Discovery 2025 – A festival of life science | Liverpool, UK: Europe’s largest drug discovery meet returns to Liverpool. Two days of talks, 200+ exhibitors and careers support; free to attend.
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