Philip Robinson

Delighted to share our paper ➡️Increased somatic mutation burdens in normal human cells due to defective DNA polymerases go.nature.com/3kUQVJy Implications for our understanding of cancer risk and the somatic mutation theory of #ageing 🧵⬇️

@JoeydeWit_ I am seeing Nume and they have solved some difficult problems in CFO copilot / AI CFO space. Worth a careful look. I heard their onboarding and integrations are 🔥.

My experience every time I try get AI to do any calculations. AI CFO's will come... but not yet.

@LifeSciVC What's perhaps even more troubling is the amount of this $300bn that is spent on repetitively building biotech infrastructure and duplicating scientific efforts.

Putting the AI capex boom from hyperscalers into context... The entire global biopharma ecosystem spent ~$300B in R&D last year to enable new medicines This year, AI capex will be 2x+ that number, just from four tech co's, to enable Skynet's Terminator

@GoogleResearch @GoogleResearch that is an erroneous claim. DeepVariant is a genome interpretation tool not a genome sequencing tool. You have not produced a genome sequencing innovation, rather a genome interpretation innovation.... Please address this claim

Sequencing a human genome, which once took 13 years and $3B, can now be done in days with the help of AI. By using AI tools like DeepVariant and DeepConsensus, we’re now helping researchers sequence the genomes of endangered species with incredible speed and accuracy. From the Grevy’s zebra to the African penguin, see how AI is helping pull species back from the brink.

@iskander Completely agree, as a fellow big data person by background. There is a lot that can be learnt about versioning and forking. Too many documents in clinical trials and the systems to manage them are not good enough. There are some people building in this area...

git for clinical trials main branch: master Protocol feature branch: sub-study merge: amend the master protocol after sub-study succeeds

@iskander @RuxandraTeslo No one is interested in taking the risk, not the patient, the drug company, the physicians. It's the first in class effect and the weight that data has. I think it isn't a bad thing necessarily, imagine how much drug development has arisen from the proceeds of Keytruda!

@drphilrobinson @RuxandraTeslo Isn’t it kinda remarkable that there are so many effectively interchangeable aPD1 drugs and yet the list prices are still >$100k / yr?

@RuxandraTeslo I think I mentioned this before but there’s an aPD1 cartel that keeps costs inflated and gets veto power over trials based on choosing who gets the drugs for free in control arms Busting this up somehow would decrease cost & improve throughput of IO trials

@DrPaulyDeSantis Very interesting analysis, impressively candid. Has the slide deck been published yet?

Biotech investors crave M&A, but it really is not an efficient use of capital most of the time. Great analysis by $REGN $IBB $XBI at #JPM26 Regeneron internal data on 450 plus biopharma deals since 2010 is sobering: -43 percent failures -Only ~10% true winners ~$350B deployed where outcomes are known -Overall IRR ~8 percent -M&A IRR 4 percent -Licensing IRR 18 percent Buying companies destroys value while buying biology selectively still works.

@MWB741 Thank you, very helpful. $GANX hasn't budged yet

@drphilrobinson That is an old PR. 79% of the 1b patients elected to continue into the extension (despite the tests, assessments, daily drinks, and lumber puncture). That's a big deal. It is clear that most of the patients experienced benefits.

$GANX — The Most Asymmetric Setup I’ve Ever Seen (And the Clock Is Ticking) This is a long post-- there's a TL;DR at the end. I’ve made some posts already about the science of GT-02287 and Parkinson’s to the best of my ability, so I won’t bore people with another one (right now). And I don’t think it is necessary to understand the science behind it to recognize all of the signs of a very promising investment. After watching Gain's CEO Gene Mack’s interview with @LouBasenese (link in comments), I was more convinced than ever that Gain Therapeutics ($GANX) could be sitting on something historic — likely the first truly disease-modifying drug for Parkinson’s. This isn’t hype. Every single data point with GT-02287 since day one has been consistent, strong, and conservative in how it’s been communicated. It's the totality of all of the pre-clinical and clinical work, the still-emerging science of Parkinson's, and company statements that make this such a compelling investment. Touching on some of his quotes in the interview. Keep in mind that he had a good read on the data by the time of this interview. 1- “We’ve seen improvements in motor function, and other sensory types of things…reports of improvements in smell, improved balance…” These are probably the most important of his quotes for obvious reasons. In 90 days, patients are showing improvements when they should be getting slightly worse. N is small, yes, but we should pay attention to what Gene is saying. He flew down to Australia to meet with the patients and the clinicians (more below). 2- “We think we have that.” Gene said: “We have to look at…what happens inside these patients so that we can tie to why they may have improved, and WE THINK WE HAVE THAT, we think we’re gonna have that. And we’ll have it by the end of the year.” That line speaks volumes. I believe they already had the data — they’re interpreting it. Parkinson’s has never been slowed or reversed before, so these biomarkers might actually represent new discoveries in neurology. Gene’s tone wasn’t speculative, IMO — it showed confidence. 3- “I think we have the first disease-modifying drug for Parkinson’s.” You don’t casually say that on record unless you’ve seen something very convincing. The way he said it — calm, grounded, and matter-of-fact — tells me he’s very confident in what the data shows. 4- “This stock is a buy at $10, it’s a buy at $15, it’s a buy at $20…Over the next two months… that should be the switch.” He’s almost laughing at how undervalued the stock price is at this level. And he should know-- for years, he was a senior analyst for multiple investment banks in the biotech and life sciences sector. This is also Gene giving a not-so-subtle hint at timing and impact: data in hand, interpretation ongoing, and release coming in December (imminent). He’s mentioned before that it will be a switch, not a dial, referring to share price. He knows what’s coming — and he’s confident enough to predict a “switch” moment, meaning the share price will explode overnight. For context, comparable sized biotechs with far less exciting data — like FULC and ANVS recently— went up over 70% overnight on their readouts. The asymmetry here is far greater. Also look at CAPR (6x overnight), PRAX (up 5X in a very short window), CADL (4x overnight in 2024), CGEN (3x overnight). The list goes on. Some of these are phase 3, but the point is that huge overnight gains happen often. And even though GT-02287 is only phase 2-ready, the historic potential here cannot be ignored. 5- “Patients were lamenting coming off the drug…” Gene said when he visited with the patients in Australia, they didn’t want to stop taking GT-02287. Clinicians felt the same — so Gain set up a 9-month extension. That’s not normal for early Parkinson’s trials. When both patients and clinicians are begging to stay on a study drug, you know something real is happening. It’s also a win-win: more long-term data for Gain, and continued benefit for participants. In response to being asked to come back after the data (and with a knowing glint in is eye): “Oh, we’re gonna have much to talk about. It’s gonna be great.” Not sure how he can more clearly communicate that he is confident in what they have without risking regulatory violation. The bigger picture: consistent wins at every step Zooming out, every milestone so far — preclinical, Phase 1a, and interim 1b — has been a home run. No safety issues, target engagement proven, mechanistic validation, functional improvement shown. Even the preclinical results (example linked in comments) were unlike anything seen before: restoring mitochondrial and lysosomal function, normalizing biomarkers, and improving motor and cognitive outcomes. The consistency across every stage has been unprecedented. Gene’s conservative nature makes his recent tone all the more meaningful — he’s never overpromised. And yet, now he’s openly saying, “I think we have the first disease-modifying drug for Parkinson’s.” This Isn't A Binary Event! The upcoming phase 1b data readout isn’t pass/fail — safety is already confirmed. The only question left is how efficacious it is. We’ve already seen encouraging UPDRS improvements, and reports of smell and balance returning. Even if the data isn’t as clean as bulls hope, the GBA1 subgroup alone (the mutation this drug was designed for) could justify a buyout at several times the current ~$250M market cap. There were three GBA1 patients in the trial, and we already saw the UPDRS scores for two of them, and they were great. The scores from one of them showed huge improvements. The drug working for the GBA1 group is my worst-case scenario at this point. There are about 100k GBA1 cases in the U.S. alone, and growing. To me, this is why the drug has been de-risked. Huge upside, little-to-no downside, IMO. Realistically, anything showing disease-modifying biomarker trends could send this stock up 100%+ overnight. And it’s not just 02287. Gain’s pipeline has other “Magellan” molecules that big pharma could develop immediately. Whoever partners or acquires them won’t be starting from scratch — they’ll be walking into a very promising platform. Gene said in the interview that some of their back-up compounds are even more potent in some cases. These include Alzheimer’s, Dementia with Lewy Bodies, Gaucher’s, Cancer, and metabolic diseases. The timing We’re now inside the likely data window — the next 6 days. Gain has had the data for weeks, Gene’s confidence has never been higher, and official company statements are telling. The company has likely been lining up interviews, media, and potential partnership/acquisition conversations. When the switch flips, it will happen fast. And this will happen soon, IMO. TL;DR - Gene Mack is not a hype CEO — yet he’s publicly telegraphing that they have the supportive data. “I think we have the first disease-modifying drug for Parkinson’s.” - Every step so far has been consistent and positive — no red flags anywhere. - The company already has safety and mechanistic proof; the upcoming biomarker data could confirm true disease modification. - $GANX is sitting at a ~ $250M market cap, with upside potential in the billions. - Data readout expected within days. This might be one of the most asymmetric setups we’ll see in biotech this decade. @kkernttb @RealAvidTrader @BiotechStockRsr @odibro @yaireinhorn @thebiotechforum @BiopharmIQ @BPharmCatalyst @SupNovaTrading @StocksPursuit @Microcapreturns @dixielee1969 @fundmyfund @makedatbread88 @SheffStation @bwsm12702 @TopStockAlerts1

@MWB741 Great analysis, the science is strong, the data is strong. I am unsure about their financial position. Low cash reserves and looking like they may become financially distressed and need to raise soon. What's your take on their runway and cash reserves?

Stem cell transplants are amazing. A patient's diseased blood is replaced by a relative or stranger's. But what does putting a blood system into a new body do to the cells? How many survive? Does it matter? Out in @Nature today, we asked some basic Qs 👇 rdcu.be/dYCoZ

Looking for PhD students/postdocs for my newly opened lab at the Institute of Human Biology of #Roche in Basel. We work on human lung, liver and gut #organoids. Send email + CV + motivation letter to hans.clevers@roche.com with header #IHB institutehumanbiology.com

Gareth Southgate’s greatest achievement was not on the pitch, but in the standard that he has set for how a role-model and representative of our country should conduct themselves. In a job which has subjected him to a degree of national scrutiny and criticism that most people will never appreciate, he has been a real mensch, remaining dignified, respectful and considerate throughout. His legacy will be a generation of young people who have learned from him that leadership is primarily about decency, integrity and bringing honour to others. That is worth more to our country than any trophy. The crown of a good name supersedes all. Thank you, Gareth.

We did an internal experiment using LLMs to parse and visualise ORRs from AACR clinical trial abstracts. Worked pretty well; check out the results in the link (click the bubbles to open the relevant abstract) aacr.convoke.bio

@sciencejannat @CellGenomics Huge Congrats @sciencejannat , this is an amaaaaazing body of work. So many hurdles overcome in this cutting-edge study!

Very excited to share that our work reconstructing chromothripsis has now been published in @CellGenomics! We reconstructed chromosomes with up to 800 rearrangements on a single allele and queried epigenetic and transcriptomic consequences. Thread below! #secsectitle0090" target="_blank" rel="nofollow noopener">cell.com/cell-genomics/…

This super paper examines the ways in which gene therapy and stem cell transplant change this delicate balance of cellular competition and explains why blood cancers arise in these patients. Big hurrah, great job @doctor_msc and colleagues !! 2/2

In the perpetual struggle for dominance, populations of cells in normal healthy tissues seek out ways to outcompete their neighbours. We know that medical treatments can alter this dynamic. 1/2

Gene therapy is big news! bbc.in/478lz7X and a major advance for patients with sickle cell disease (SCD) & thalassemia. But in some recent trials, a small number of patients developed blood cancer. Our study (out in @NatureMedicine ) asked why... nature.com/articles/s4159…

🚨For the 4th day in a row, the @IDF opened an evacuation corridor for civilians in northern Gaza to move southwards. Since 10:00 am today, thousands of Gazans passed through. The corridor will be open until 14:00 so that Gazans in the north can evacuate for their safety.

The IDF is facilitating the delivery of humanitarian aid to Gazan civilians through @cogatonline COGAT’s Spokeswoman Shani Sasson shares more details: