Rob Shaffer

Truth.

Perspective by Kevin Schulman, MD, and Arthur L. Kellermann, MD, MPH: Substandard Generic Drugs — Threats to Patient Safety and National Security nejm.org/doi/full/10.10… #HealthPolicy #PublicHealth

Interesting enzymatic approach to clearing amyloid for AD from an Ultragenyx spinout. AAV9 delivered PPCA enzyme. Amlogenyx Announces Positive Preclinical Data on AM805, a Potent Amyloid-Degrading Protease for the Treatment of Alzheimer’s Disease amlogenyx.com/wp-content/upl…

Data look quite good. My Q's: 1) Will murine activity predict that in NHPs, humans, and why? 2) What about heart? Bayer must have thought they have good cardiac data given the announced deal. 3) Is it enough differentiation to be more potent, if it's still given IV?

Thought this was a good article for those looking to position themselves well for a future CEO role. BCG | From COO to CEO: Are You Ready to Make the Leap? on.bcg.com/4rPtNfn via @BCG

@Biohazard3737 Yes and OneNote. I don't know why they even have a search bar given its lack of ability to search

Make the FDA Sophisticated Again

Small upfront, big biobucks, very interesting deal Tenaya Therapeutics Enters into Research Collaboration with Alnylam Pharmaceuticals to Identify and Validate Novel Genetic Targets for Cardiovascular Disease Therapeutics globenewswire.com/news-release/2…

This report describes the effects of zorevunersen, an antisense oligonucleotide designed to up-regulate NaV1.1 sodium channels, in patients with Dravet syndrome, which is a severe developmental and epileptic encephalopathy. Read the full report: nejm.org/doi/full/10.10… Science behind the Study: Toward a Disease-Modifying Therapy for Dravet Syndrome nejm.org/doi/full/10.10…

Ah man. From PepGen: "U.S. FDA places FREEDOM2 trial [for DM1] on partial clinical hold related to preclinical pharmacology and toxicology" PepGen Announces Regulatory Updates on FREEDOM2 finance.yahoo.com/news/pepgen-an… $PEPG

Novartis closes Avidity deal as rare heart disease spinout launches - endpoints.news/novartis-close…

Atrium Therapeutics Launches with Approximately $270 Million to Advance Novel RNA Medicines for Rare Genetic Cardiomyopathies finance.yahoo.com/news/atrium-th…

Avidity's cardiology SpinCo launches, seems well-capitalized. PRKAG2 program in the clinic 2H26 and PLN IND-enabling this year, with IND next year. I guess BMS didn't bite (I believe they had a ROFR/N on these assets from their collaboration with Avidity)

Infuriating

Seems like Aro is pivoting toward autoimmune and away from rare genetic disease? I always thought targeting GYS1 for Pompe was a little odd for their first program Aro Biotherapeutics expects to double headcount after strategy shift bizjournals.com/philadelphia/n…

RFK Jr. touts “removing mercury” from vaccines, when in reality it wasn’t even elemental mercury, it was nontoxic, it was safety tested, and it had already been removed from every routine shot. Meanwhile, the same admin is clearing the way for ACTUALLY MERCURY IN ITS TOXIC FORM.

Major life hack: Be optimistic. The way you choose to perceive the world impacts every single area of your life. Choose wisely.

[Potentially Historic/Destructive Storm southeast of the BOS-PVD corridor] We are very concerned for areas southeast of the Boston-Providence corridor where wet snow combines with northeast wind gusts of 60-70 mph. Power outages & tree damage. Worst conditions Mon 4 am to noon.

Antibody-oligo conjugates work. Here, Avidity's del-desiran (now Novartis $nvs)