@newtonianmade.bsky.social
Decent drug developer for over 10 years; decent father for over 12 years; decent husband for over 15 years; overall decent guy for over 50 years
We need more of this. Thank you Bruce.
music.apple.com/us/album/str...
This data is fascinating. So positive. So much progress. Why again are we not continuing to fund science???
www.cancer.org/research/acs...
I really don’t understand why people accept variability and self-determination in physical features like eye color or hair color or muscles. People accept variability and self-determination in interests like education or music. But some can’t accept variability and self-determination in gender.
15.01.2026 00:17 — 👍 0 🔁 0 💬 0 📌 0It's great to see this dedication to patients. Spinraza was first approved in 2016 and now a higher dose is approved. Data most clear for NfL but function has followed improvement in NfL in SMA.
#JPM #biotech $BIIB
investors.biogen.com/news-release...
When do you think this will be implemented?
13.01.2026 12:53 — 👍 0 🔁 0 💬 0 📌 0
Alnylam has more opportunities than they have resources to capitalize. I believe the coming years could demonstrate the power of the siRNA modality.
#JPM2026 #biotech
investors.alnylam.com/press-releas...
I have followed Ionis for a long time. It feels like they have turned a corner and are beginning to capitalize on the science they have pioneered.
#JPM #biotech
ir.ionis.com/news-release...
Happy JPM to all who observe.
11.01.2026 18:49 — 👍 1 🔁 0 💬 0 📌 0Is there such a thing as a 12-alarm fire? This Perspective piece is scathing and absolutely 100% on point.
04.12.2025 00:59 — 👍 0 🔁 0 💬 0 📌 0
12 former commissioners of the FDA came together to write a Perspectives piece for the New England Journal of Medicine; raising our concerns about recent changes to vaccine approval policy at the FDA and its implications for patients and public health.
03.12.2025 22:28 — 👍 619 🔁 270 💬 9 📌 11
Rosalind Franklin, 1920-1958
en.wikipedia.org/wiki/Rosalin...
Last night, Sarepta $SRPT announced 3rd quarter results. They were not good. A major decline in GTx revenue. They also announced that the confirmatory study for 2 PMOs was not positive after 96 wks. 3 PMOs confirmatory trials have now failed.
#sarepta
investorrelations.sarepta.com/news-release...
I am thoroughly enjoying Breath from Salt, the story of cystic fibrosis. From its origins of misdiagnosis to innovative treatments, this story resonates with me. I highly recommend this book.
#biotech #cysticfibrosis
Do you think the ISS should be scrapped or is there a way to turn it into something more?
02.11.2025 12:43 — 👍 0 🔁 0 💬 1 📌 0
What a great result for patients! A profound improvement in hearing for patients after administration in a contained space that limited any potential safety concerns.
investor.regeneron.com/news-release...
It seems like so much of the conversation is about what we are giving up as we move into the future, what about everything we can gain?
Green energy jobs, biotechnology cures, so many possibilities when we work together.
I like this quote from Winston Churchill
#biotech #persistence
Newborn screening is an unappreciated benefit of modern society. The ability to diagnose people at birth with diseases so that they can be treated or perhaps participate in clinical trials benefits individuals and all of society.
#biotech #biosky #newbornscreening
endpoints.news/the-trump-ad...
Tough news for Capricor.
They received a CRL on Friday. Approval on a single trial with new technology and without dramatic benefit was always going to be difficult. New leadership at FDA likely wasn't helpful.
Good news is that another trial is already underway.
www.capricor.com/investors/ne...
Good news for patients. Biogen announced today that they are moving salanersen to P3. Spinraza has been a blockbuster for Biogen and for patients.
Development of salanersen has occurred because of neurofilament as a biomarker.
#biotech #biosky #SMA #Biogen
investors.biogen.com/news-release...
When should the safety profile of an immunosuppression regimen really become the safety profile for the drug?
One wouldn't have events without the regimen, and they wouldn't have to take the regimen without taking the drug.
This may come up again with GTx where Sarepta is talking about a regimen.
Good news for patients. Over the weekend, $VRTX announced good data for Zimislecel from their Phase 1/2 trial for T1DM.
12/12 participants achieved HbA1c <7% and safety profile was similar to immunosuppression.
#biotech #biosky
investors.vrtx.com/static-files...
Good news for patients. Compass Pathways $CMPS reports positive data on Psilocybin for Treatment-Resistant Depression showing statistically significant benefit with a reasonable safety profile.
The drug is also being tested for PTSD and anorexia
#biotech #biosky
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This is good news for patients. More data showing that it is possible to preserve muscle while addressing adiposity.
Also, confirmational data of biologic mechanism for apitegromab that may be useful in other indications.
#biotech #biosky #sarcopenia
investors.scholarrock.com/news-release...
Dyne provided an update on their DM1 program data and regulatory strategy. The safety and efficacy data continues to look as good or better than Avidity.
DM1 is large enough for multiple winners, especially one that addresses CNS.
#biotech #biosky #FDA
investors.dyne-tx.com/static-files...
I know I am posting late to this story, but it still needs to be discussed.
I believe gene therapy in a contained space may be safe and effective. But weight-based dosing leading to significant exposure doesn't appear safe and may not be effective.
investorrelations.sarepta.com/news-release...
Gene therapy in contained spaces may work and may have value as demonstrated by the SpliceBio news below.
Benitec, $BNTC, is also interesting. They are injecting gene therapy into specific muscles in OPMD, a very rare form of muscular dystrophy.
#biotech #biosky
splice.bio/splicebio-se...
Another from New Amsterdam, $NAMS, shows improvement in p-tau217, a known biomarker for AD.
#biotech #biosky
ir.newamsterdampharma.com/news-release...
Biomarkers are key to 2 interesting results reported today. One from Avidity, $RNA, shows improvement in a new biomarker for FSHD and possible stabilization or less decline in function.
#biotech #biosky
aviditybiosciences.investorroom.com/2025-06-09-A...
