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GeneDoctor

@GeneDoctorB

Biotech Investing - Genetic Medicine, Rare Disease, Translational Innovation, AI-Powered Target Discovery and Validation, Not Investment Advice

Joined June 2024
  • Tweets65
  • Following66
  • Followers280
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Photo by GeneDoctor (@GeneDoctorB)Photo by GeneDoctor (@GeneDoctorB)Photo by GeneDoctor (@GeneDoctorB)Photo by GeneDoctor (@GeneDoctorB)Photo by GeneDoctor (@GeneDoctorB)Photo by GeneDoctor (@GeneDoctorB)Photo by GeneDoctor (@GeneDoctorB)Photo by GeneDoctor (@GeneDoctorB)Photo by GeneDoctor (@GeneDoctorB)Photo by GeneDoctor (@GeneDoctorB)
And the answer is: eplontersen mean KD 69%, median 76% (cardioTTRansform) vs. vutrisiran mean KD 81%, median 87% (HELIOS-B). Nucresiran will be even better in TRITON-CM!
Image from John Maraganore 🇺🇸🇬🇷🇺🇦 (@JMaraganore)
John Maraganore 🇺🇸🇬🇷🇺🇦 (@JMaraganore) profile pictureJohn Maraganore 🇺🇸🇬🇷🇺🇦
@JMaraganoreAug 28
Bottom line… NOT all “silencers” created equal!
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Vinay Prasad Is Out at the FDA—Again wsj.com/opinion/fda-marty-ma…
Opinion | Vinay Prasad Is Out at the FDA—Again

Opinion | Vinay Prasad Is Out at the FDA—Again

The high-handed rejection of a treatment for Huntington’s disease shows why the biologics chief had to go.

wsj.com
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“The FDA official responded, “You all know that as background, I’m a professor and I’m a practicing [hematology-oncology] doctor. I do miss my clinic and I miss teaching, and I miss being able to run my podcast and speak freely.” statnews.com/2026/03/06/fda-…
The FDA, urged to avoid controversy, creates a new headache with attack against UniQure

The FDA, urged to avoid controversy, creates a new headache with attack against UniQure

Recent decisions at the FDA threaten to turn its disagreements with the drugmaker UniQure into another political headache for the Trump administration.

statnews.com
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BRAVO!
Alnylam Pharmaceuticals (@Alnylam) profile pictureAlnylam Pharmaceuticals
@AlnylamJan 11
We've unveiled our new five-year strategy and shared preliminary Q4 and FY2025 global net product revenues, 2026 pipeline goals and product sales outlook. Read the press release: bit.ly/4jxw4IU Join our live #JPM26 webcast tomorrow at 9 AM PT: bit.ly/4srb8ax
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GeneDoctor retweeted
Eric Topol (@EricTopol) profile picture
Eric Topol
@EricTopol24 Dec 2025
For Alzheimer's disease, it's no longer a debate of amyloid (Aβ) versus tau. They are interactive drivers and this new, optimistic review maps out a path towards disease-halting therapies @CellCellPress cell.com/cell/fulltext/S0092…
Image from Eric Topol (@EricTopol)
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Super cool @zhangf!
Feng Zhang (@zhangf) profile pictureFeng Zhang
@zhangf17 Dec 2025
Excited to share our new work on immune aging! We explored whether the liver could serve as a temporary "factory" to produce immune factors that decline with aging, potentially helping to rejuvenate aged immunity. @mircoscopy.
Image from Feng Zhang (@zhangf)
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I’m the former head of Pfizer R&D. I’m very worried about biopharma’s future statnews.com/2025/12/19/phar… via @statnews
I’m the former head of Pfizer R&D. I’m very worried about biopharma’s future

I’m the former head of Pfizer R&D. I’m very worried about biopharma’s future

The New York Times once called John Lamattina, then Pfizer head of R&D, “Dr. Optimistic.” He’s not so optimistic now.

statnews.com
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Good to see these type of combos to build critical mass in areas like rare disease!
Adam Feuerstein ✡️ (@adamfeuerstein) profile pictureAdam Feuerstein ✡️
@adamfeuerstein19 Dec 2025
Replying to @adamfeuerstein
biomarin.com/news/press-rele…

BioMarin to Acquire Amicus Therapeutics for $4.8 Billion, Expanding Position as a Leader in Rare...

biomarin.com
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“For decades, the U.S. has been the pinnacle of global science. Now experts fear it may have lost some of its luster, and that budding international researchers won’t… take their next career step in the U.S.” Not MAGA or MAHA.
Damian Garde (@damiangarde) profile pictureDamian Garde@damiangarde17 Dec 2025
Amid talk of a brain drain, some scientists leave U.S. behind statnews.com/2025/12/17/rese…
Amid talk of a brain drain, some scientists leave U.S. behind

Amid talk of a brain drain, some scientists leave U.S. behind

“A large part of what we loved about the United States is no longer there”: Amid talk of a brain drain, some scientists leave the U.S. behind.

statnews.com
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GeneDoctor retweeted
Eric Topol (@EricTopol) profile picture
Eric Topol
@EricTopol21 Aug 2025
A discovery of a gene underpinning the perception of pain with implications for treating the chronic condition that affects 600 million people globally economist.com/science-and-te… nature.com/articles/s41586-0…
The discovery of a gene for chronic pain could herald new treatments

The discovery of a gene for chronic pain could herald new treatments

Even diet might have an effect

economist.com
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A major milestone for genetic medicines! First-ever in vivo genetic correction of a human disease! Amazing result for science, medicine, and patients! So proud of the ⁦Beam team! globenewswire.com/news-relea…
Beam Therapeutics Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1...

Beam Therapeutics Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1...

Single Dose of BEAM-302 Led to Durable, Dose-dependent Increases in Total and Functional Alpha-1 Antitrypsin (AAT), Production of Corrected M-AAT, and...

globenewswire.com
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GeneDoctor retweeted
xTrade ⚛ (@bill_us) profile picture
xTrade ⚛
@bill_us7 Mar 2025
$SGMO. Recalled $PFE handed $ROIV biotech gift of year in 2023, think they might do it again in 2025, this time for $SGMO. If both Fabry and HemoA deals go through for $SGMO, it could be a $10 stock.
Phoenix Biotech (@BiotechAnalysst) profile picturePhoenix Biotech@BiotechAnalysst16 Jan 2025
Barclays: $SGMO says inbound interest for #HemoA GT program and would transfer the program from $PFE to new partner Hemo GT #UPDATE here likely #April #Fabry deal announcement likely in Q1 #competitive #bidding upfront payment ~ $250M , milestones and double digit royalties
Image from Phoenix Biotech (@BiotechAnalysst)
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The biggest problem of Gene Editing & Gene therapy is delivery - how to deliver a genetic payload to the exact desired location. Here’s a great Infograph which shows which BioTech companies use Viral delivery methods (AAVs, LVs) compared to non-viral platforms (NLPs). $XBI
The biggest problem of Gene Editing & Gene therapy is delivery - how to deliver the genetic payload to the exact desired location. Here is a great Infograph which shows which BioTech companies use Viral (AAVs) delivery methods compared to non-viral platforms (NLPs).

ALT The biggest problem of Gene Editing & Gene therapy is delivery - how to deliver the genetic payload to the exact desired location. Here is a great Infograph which shows which BioTech companies use Viral (AAVs) delivery methods compared to non-viral platforms (NLPs).

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Surrogate endpoints for potential #accelerated #approval viewed as much more straightforward for biologics, particularly #gene #therapies $SGMO FABRY $SRPT DMD $RGNX MPS
Image from Phoenix Biotech (@BiotechAnalysst)
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