Rob Shaffer

Truth.

The paradox of biotech protectionism: Why walling off China biotech weakens America US ban on Chinese biotech/trials would return pharma leadership to Europe, slow US patient access to new meds, & lead to US dependency protectionism claims to prevent. rapport.racap.com/all-stories/th…

Of course. Current gen AOCs go to many tissues, so there's some optionality. siRNA is potent enough that as long as that payload is specific, you've got a viable drug candidate. Novartis says Avidity's science could apply beyond muscular dystrophies - endpoints.news/novartis-says-…

Just added bsAbs, mRNA, and oligo estimators (ASO + siRNA) to biologics.tools 2 surprises I did not know: 1) ds siRNA actually slightly less $/g than ss ASO due to cheaper "typical" 2' chemistry. 2) LNP formulation is roughly same cost as making the mRNA DS Still free!

@well_beck @daphnezohar Thanks! That's correct. Just a matter of scope; could imagine adding regulatory costs, tox studies, engineering runs, and on and on and on. Maybe stuff that could be added in the future

@ShafferBiotech @daphnezohar Amazing tool! It does not appear to capture costs of analytical development from discovery through commercial though and please feel free to dm me for insights into that for each of the different modalities.

Built a free biologics COGS estimator covering mAbs, recombinant proteins, ADCs, and C&GT, with GMP/Tox/RG options. For founders, investors, and BD folks who need a defensible cost figure for pitching/evaluating. Feedback welcome; still iterating! biologics.tools

@Nicole_Paulk Thanks so much @Nicole_Paulk ! Please do share with them!

@ShafferBiotech This is so cool Rob I LOVE it!!! I've got a bunch of #RareDisease groups I'm going to send this to!!!! This is awesome 😎

@mtzi5 Try again, it's working. I think it went down for a bit. Let me know if it's still not working

@ShafferBiotech Link doesn’t seem to work- would be interested to test

@anthonystaj Try again, it's working. I think it went down for a bit. Let me know if it's still not working

@ShafferBiotech sounds cool, website doesn't work though

@PearlF 🙏 thank you Pearl

A biotech founder/COO vibe coded a free tool 4when small biotechs need production cost figures that are defensible—transparent COGS estimator for mAbs, recombinant proteins, ADCs, & C&GT & 3 manufacturing grade tiers (GMP, Preclinical/Tox, Research). He's looking for feedback ⬇️

Asked mid-season about the Yankees, former Red Sox General Manager Theo Epstein once said that he had looked at their roster at the start of the season, realized that they'd win 100 games, and now ignored their weekly standings in order to focus on his own roster's ability to compete. I raised this anecdote at a recent roundtable of experts discussing China's rise in biotechnology. China is uplifting scientists, investing hundreds of billions in R&D, and accelerating clinical trials. It's going to win 100 games. The center of biomedical gravity is moving to China from America, just like it moved from Europe to America at the close of last century. Unless America decides to win 110 games. statnews.com/2026/05/18/bio…

I'm a middle eastern historian. My own family were made refugees. And this is my honest view of the Nakba (“catastrophe”) - the displacement of around 700,000 Palestinian Arabs during the 1947–49 war surrounding the creation of Israel. A thread. 🧵

Great news re: new health insurance reforms—MA Governor Healey announced that Division of Insurance finalized regs that eliminate prior authorization requirements for routine & essential healthcare incl cancer scans & medications for chronic conditions like asthma, diabetes & heart disease. More below:

Excited to debut data this week at #ASGCT26 that my startup company @HydrogeneTx has been working on in stealth. We have achieved non-viral DNA delivery into NHP liver at levels matching commercial AAV products for hemophilia. Oral presentation tomorrow! prnewswire.com/news-releases/…

CPPs <<< receptor-targeted approaches

Oral smolecule inhibitor of GYS1 originally developed by Maze Tx --> Shionogi Announces First Patients Enrolled in Global Phase 2 Clinical Trial in Adults with Late-Onset Pompe Disease shionogi.com/global/en/news…

I continue to fear for the future of US competitiveness. While not a true zero-sum game, harming our scientific capabilities will benefit our adversaries. Immigration changes are driving foreign researchers to leave the U.S. — or not come to begin with  statnews.com/2026/05/04/tru…

@Noblepariah888 @plainyogurt21 Tried multiple systems, solubility tags. Was a decade ago so maybe expression systems have improved enough by now, not sure

@ShafferBiotech @plainyogurt21 Why was that? Were you doing E.Coli plasmid transformation?

Why doesn't every enzyme deficiency disease have an ERT?

@Noblepariah888 @plainyogurt21 Biomanufacturing process

@ShafferBiotech @plainyogurt21 "MTM1 doesnt like to express" express in the biomanufacturing process or in the body?

Likely. One ex: I was involved with a previous co trying to make ERT for XLMTM. A fusion protein of MTM1 and a carrier to get it in cells. Great dz model mouse data, but MTM1 just doesn't like to express. Not a viable program. Then you get into, well how can you get it in cells, and CPPs are garbage, so can you use TfR or something similar? Maybe! Then you get into other delivery questions like tissue specificity. Oh and nAbs/ADAs are a big deal. If the patient doesn't express the enzyme at all, they are likely to mount a response. How serious of a response is tolerable?

Capital problem for ROI? There are billion dollar companies built on the backs of rare disease ERT and they are money printing machines. Surprised no one has made this investment. DNLI is probably the closest example of one using their BBB platform for brain delivered enzymes but they still miss things like PMM2

@plainyogurt21 Former. Very challenging in many cases but agreed

@ShafferBiotech engineering problem or biology problem? the former is solvable