Rob Shaffer

Biotech entrepreneur and consultant. Biochem PhD. Opinions strictly my own.

[Rob Shaffer @ShafferBiotech]

Truth.

[Rob Shaffer @ShafferBiotech]

Beautiful evening at Fenway

[Bruce Booth @LifeSciVC]

Patient-first, America-first: The Case For Global Collaboration.
While the rise of Chinese biopharma in the past decade has been meteoric, I’m firmly in the camp that their engagement in the global ecosystem is good for the sector – its more opportunity than threat, great for patients, and is a healthy forcing function to raise the game for everyone
New blog link 👇

[steve usdin @steveusdin1]

Pharma CEO silence is complicity in the destruction of U.S. science.

Office of Management and Budget Director Russell Vought has poured gas on the foundations of science. Pharma CEOs should demand that President Donald Trump take away the matches.

For more than a year, the Trump administration has been weakening the institutions that made the United States the global leader in biomedical innovation. This week, Vought revealed that the objective is not merely to shrink the federal research enterprise, but to make it subservient to political and ideological objectives.

Vought’s proposed rule would require senior political appointees to review and approve every discretionary federal grant before it is awarded, explicitly barring them from deferring to peer reviewers, and would mandate that all grant programs align with the president’s policy priorities, rather than scientific need or expert consensus. It seeks to introduce ideological tests on applicants and institutions that receive grants, bar most international scientific collaboration, and allow political appointees to cancel grants at any time without providing an explanation. The proposal also would allow agencies to make grants that haven’t been publicly announced, opening the process to corruption and favoritism.

If implemented, the proposal would represent the most serious assault on U.S. science since World War II. Even those who agree with the Trump administration’s views on social and political issues should be alarmed. Future administrations with very different views may not abandon the temptation to wield control over scientific funding to advance ideological agendas.

More at https://t.co/TA0gCF82gR (no paywall).

[Daphne Zohar @daphnezohar]

A few people have asked me to comment on this article & the topic of US biotech competitiveness vis-à-vis China. I agree w/ Peter & Tess that expanding the COINS Act to biotech would have negative consequences. Risks handing global leadership & profits to EU competitors while doing little to slow China’s dominance. Also agree that we must protect critical supply chains/manufacturing from a national security perspective.

While COINS for biotech is a bad idea, we need proactive legislation to strengthen US biotech innovators. Targeted measures could include tradable/refundable R&D tax credits (noted in article) & priority review vouchers (@rtnarch), NOL reforms (amending Section 382), enhanced data exclusivity for genuine first-in-class assets & patent reform. These could level the playing field for US innovators without the protectionism & restrictions that would raise costs & slow patient access to new medicines

[Peter Kolchinsky @PeterKolchinsky]

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.

https://t.co/DJAkWQfd5X

[Rob Shaffer @ShafferBiotech]

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 - https://t.co/ByAvK0rk1n

[Rob Shaffer @ShafferBiotech]

Just added bsAbs, mRNA, and oligo estimators (ASO + siRNA) to https://t.co/yVkJwN2YtJ

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!

[Rob Shaffer @ShafferBiotech]

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!

https://t.co/GqVCBliaro

[Rob Shaffer @ShafferBiotech]

@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

[Rob Shaffer @ShafferBiotech]

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!

https://t.co/GqVCBliaro

[Rob Shaffer @ShafferBiotech]

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

[Rob Shaffer @ShafferBiotech]

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

[Rob Shaffer @ShafferBiotech]

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

[Rob Shaffer @ShafferBiotech]

@PearlF 🙏 thank you Pearl

[Rep. Jake Auchincloss 🟧 @RepAuchincloss]

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.

https://t.co/8H4eCKOzb8

[SK Tedeschi @skedeschi]

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. 🧵

[Robert Kruse, MD, PhD @RobertLKruse]

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!

https://t.co/7mvUECiqs8

[Rob Shaffer @ShafferBiotech]

CPPs <<< receptor-targeted approaches

[Jason Mast @Jasonmmast]

Next-gen Duchenne drug from $TRDA disappoints

Startup was competing against $DYNE, $NVS and others to develop new exon skippers and other muscle-targeted drugs

https://t.co/Tnc7CbdO0c

[Rob Shaffer @ShafferBiotech]

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 https://t.co/fmfB7UiQDQ

[Rob Shaffer @ShafferBiotech]

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 

https://t.co/MUOQETv1FE