@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
Is there such a thing as a 12-alarm fire? This Perspective piece is scathing and absolutely 100% on point.
Twelve former commissioners of the FDA express concern that the agency’s recent moves will undermine a regulatory model designed to ensure vaccine safety, effectiveness, and availability. Read the full Perspective: nej.md/48Ova76
December 4, 2025 at 12:59 AM
Is there such a thing as a 12-alarm fire? This Perspective piece is scathing and absolutely 100% on point.
Reposted
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.
December 3, 2025 at 10:28 PM
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.
Reposted
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...
#sarepta
investorrelations.sarepta.com/news-release...
November 4, 2025 at 12:31 PM
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...
#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
#biotech #cysticfibrosis
November 4, 2025 at 12:22 PM
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
#biotech #cysticfibrosis
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...
investor.regeneron.com/news-release...
October 13, 2025 at 11:54 AM
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...
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.
Green energy jobs, biotechnology cures, so many possibilities when we work together.
September 28, 2025 at 7:00 PM
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.
Green energy jobs, biotechnology cures, so many possibilities when we work together.
September 22, 2025 at 12:13 AM
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...
#biotech #biosky #newbornscreening
endpoints.news/the-trump-ad...
The Trump administration disbanded a newborn screening panel. Advocates now face a harder path
atient groups face a harder and unpredictable path going state-by-state to boost screening for rare but treatable conditions after the Trump administration disbanded a federal advisory committee on ne...
endpoints.news
July 16, 2025 at 1:31 PM
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...
#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...
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...
July 14, 2025 at 12:37 PM
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...
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...
Development of salanersen has occurred because of neurofilament as a biomarker.
#biotech #biosky #SMA #Biogen
investors.biogen.com/news-release...
June 25, 2025 at 1:55 PM
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...
Development of salanersen has occurred because of neurofilament as a biomarker.
#biotech #biosky #SMA #Biogen
investors.biogen.com/news-release...
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...
12/12 participants achieved HbA1c <7% and safety profile was similar to immunosuppression.
#biotech #biosky
investors.vrtx.com/static-files...
June 24, 2025 at 1:26 PM
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...
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
s204.q4cdn.com/927483861/fi...
The drug is also being tested for PTSD and anorexia
#biotech #biosky
s204.q4cdn.com/927483861/fi...
June 23, 2025 at 12:49 PM
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
s204.q4cdn.com/927483861/fi...
The drug is also being tested for PTSD and anorexia
#biotech #biosky
s204.q4cdn.com/927483861/fi...
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...
Also, confirmational data of biologic mechanism for apitegromab that may be useful in other indications.
#biotech #biosky #sarcopenia
investors.scholarrock.com/news-release...
investors.scholarrock.com
June 18, 2025 at 6:31 PM
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...
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...
DM1 is large enough for multiple winners, especially one that addresses CNS.
#biotech #biosky #FDA
investors.dyne-tx.com/static-files...
investors.dyne-tx.com
June 18, 2025 at 5:59 PM
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...
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...
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...
investorrelations.sarepta.com
June 18, 2025 at 5:54 PM
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...
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...
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...
SpliceBio Secures $135 Million Series B Financing to Advance Lead Program SB-007 in Stargardt Disease and Expand Pipeline of Genetic Medicines - Splice Bio
SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today announced the close of a $135 million Series B financing...
splice.bio
June 11, 2025 at 1:25 PM
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...
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...
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...
#biotech #biosky
aviditybiosciences.investorroom.com/2025-06-09-A...
Avidity Biosciences Announces Positive Topline Phase 1/2 FORTITUDE™ Data Demonstrating Consistent Improvement Across Multiple Functional Measures Compared to Placebo in Del-Brax Treated FSHD Participa...
-- Unprecedented data from FORTITUDE™ dose escalation cohorts for del-brax treated participants, compared to placebo, demonstrate improvement in function, strength and PROs as well as rapid and...
aviditybiosciences.investorroom.com
June 9, 2025 at 5:37 PM
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...
#biotech #biosky
aviditybiosciences.investorroom.com/2025-06-09-A...
Reposted
A post on innovation and invention in #BigPharma by @yacineazzouz (not on #Bluesky). I am not sure that I 100% agree with the placement of some of these companies.
June 6, 2025 at 8:32 AM
A post on innovation and invention in #BigPharma by @yacineazzouz (not on #Bluesky). I am not sure that I 100% agree with the placement of some of these companies.
Digital assessments are finally here. With AI and NHx comparisons, we now can appropriately interpret the data.
In addition to better evaluations of real-world environments, digital assessments create better compliance. Here is an example from DMD for SV95C.
#Biotech
hs.sysnav.com/hubfs/MDA_20...
In addition to better evaluations of real-world environments, digital assessments create better compliance. Here is an example from DMD for SV95C.
#Biotech
hs.sysnav.com/hubfs/MDA_20...
June 5, 2025 at 12:54 PM
Digital assessments are finally here. With AI and NHx comparisons, we now can appropriately interpret the data.
In addition to better evaluations of real-world environments, digital assessments create better compliance. Here is an example from DMD for SV95C.
#Biotech
hs.sysnav.com/hubfs/MDA_20...
In addition to better evaluations of real-world environments, digital assessments create better compliance. Here is an example from DMD for SV95C.
#Biotech
hs.sysnav.com/hubfs/MDA_20...
This is a big step!
Who knows what other agencies will say but for the FDA to actually say that a platform approach is possible will make more rare indications drugable.
Other sponsors will now follow.
#FDA #biotech $SRPT
investorrelations.sarepta.com/news-release...
Who knows what other agencies will say but for the FDA to actually say that a platform approach is possible will make more rare indications drugable.
Other sponsors will now follow.
#FDA #biotech $SRPT
investorrelations.sarepta.com/news-release...
U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta’s Investigational Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophy Type 2E/R4 | Sarepta Th...
CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jun. 4, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the rAAVrh74 viral vector ...
investorrelations.sarepta.com
June 4, 2025 at 2:50 PM
This is a big step!
Who knows what other agencies will say but for the FDA to actually say that a platform approach is possible will make more rare indications drugable.
Other sponsors will now follow.
#FDA #biotech $SRPT
investorrelations.sarepta.com/news-release...
Who knows what other agencies will say but for the FDA to actually say that a platform approach is possible will make more rare indications drugable.
Other sponsors will now follow.
#FDA #biotech $SRPT
investorrelations.sarepta.com/news-release...
This figure from this nice review article of GLP-1 based therapies is a nice reminder that there is always more that can be done.
Ways to innovate on basic understandings and improve outcomes for patients will continue to drive drug development.
#obesity #biotech
www.nature.com/articles/s41...
Ways to innovate on basic understandings and improve outcomes for patients will continue to drive drug development.
#obesity #biotech
www.nature.com/articles/s41...
June 4, 2025 at 12:42 PM
This figure from this nice review article of GLP-1 based therapies is a nice reminder that there is always more that can be done.
Ways to innovate on basic understandings and improve outcomes for patients will continue to drive drug development.
#obesity #biotech
www.nature.com/articles/s41...
Ways to innovate on basic understandings and improve outcomes for patients will continue to drive drug development.
#obesity #biotech
www.nature.com/articles/s41...
I hope this works but I am skeptical.
It could be really helpful to reduce the time required for menial tasks like finding documents and sorting data.
But it could also be really dangerous if it is used to make safety or efficacy decisions alone.
#FDA #biotech
www.fda.gov/news-events/...
It could be really helpful to reduce the time required for menial tasks like finding documents and sorting data.
But it could also be really dangerous if it is used to make safety or efficacy decisions alone.
#FDA #biotech
www.fda.gov/news-events/...
FDA Launches Agency-Wide AI Tool to Optimize Performance for the American People
FDA Launches Agency-Wide AI Tool
www.fda.gov
June 3, 2025 at 12:48 PM
I hope this works but I am skeptical.
It could be really helpful to reduce the time required for menial tasks like finding documents and sorting data.
But it could also be really dangerous if it is used to make safety or efficacy decisions alone.
#FDA #biotech
www.fda.gov/news-events/...
It could be really helpful to reduce the time required for menial tasks like finding documents and sorting data.
But it could also be really dangerous if it is used to make safety or efficacy decisions alone.
#FDA #biotech
www.fda.gov/news-events/...