Alexey Bersenev

23.1K posts

Alexey Bersenev

Alexey Bersenev

@cells_nnm

Cell trialist.

New Haven, CT Katılım Mart 2008
325 Takip Edilen5.7K Takipçiler
Gene Investing w/Anthony 🧬
Don’t even own it currently and I can say this market is mentally retarded when it comes to gene editing. $BEAM $NTLA $PRME A chimpanzee would have a higher IQ. Beam just showed the best ever data by far in AATD, which is a large market, and it goes from $27 pre-market to $23+. I’d love to talk to the market and ask it why it has no brain cells.
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Wei Zhao
Wei Zhao@zhaoweiasu·
$PRME plans to submit a BLA following final regulatory alignment. If all goes well, PM359 will be the first FDA-approved gene correction and 2nd or 3rd CRISPR-based gene editing therapy. CGD NCF1 deltaGT is an ultra-rare disease/mutation, and PM359 checked all the boxes of the FDA's latest draft guidance on Targeted Individualized Therapies 1⃣Identifying the disease-causing abnormality✅ 2⃣Targets the root cause or proximate biological pathway✅ 3⃣Relying on well-characterized natural history data in untreated patients✅ 4⃣Confirming successful target drugging or editing✅ 5⃣Demonstrate improvement in clinical outcomes, disease course, or biomarkers✅
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Dr. Marty Makary
Dr. Marty Makary@DrMakaryFDA·
Happy to announce today regulatory flexibilities for cell and gene therapies. Patients and those working on cures deserve common sense approaches.
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Andrew Pannu
Andrew Pannu@andrewpannu·
BMS just spent $1.5B on Orbital Therapeutics. Here's what the in vivo cell therapy landscape looks like now: I used @sleuthinsights to aggregate 113 assets and mapped 107 across 51 select players. Some takeaways: First, a statistical breakdown of the landscape: • Stage: 86% clinical vs. 14% preclinical • TA: 58% oncology, 17% I&I, 18% both, 8% other • Geo: 54% US, 35% China, 5% Both, 6% Other • Targets: 30% CD19, 37% Other, 32% N/A The BMS acquisition is the latest in a string of Pharma interest: • ABBV / Capstan ($2.1B) • AZN / Esotec ($1B) • GILD / Interius ($350M) Autoimmune disease has been in focus across these deals - and for good reason. Despite high annual costs ($60-80K per year), most patients don't respond or only partially respond to existing treatments And since Pharma always needs more blockbusters, it's easy to see the commercial appeal: • 100+ autoimmune diseases affect 15-20M Americans (4.5% globally), with rising incidence due to lifestyle / environmental factors and improved diagnosis • Unlike most cancers, I&I patients can be young, meaning a near-curative therapy option has a lifetime of benefit and cost savings • It's a massive market with $100B in aggregate economic burden; for context, the US has 80K annual NHL cases vs. >1M SLE cases The space is still very early, and based on management comments, these deals primarily provide platform optionality. Compared to ex vivo cell therapies, an in vivo approach can be safer, more scalable and more convenient, while still offering a potential functional cure. The safety bar is much higher in AID than oncology and there are a lot of options (including many generics) to cycle through already, so the playbook is to demonstrate efficacy in the most severe, non-responding patients first and build up data to progressively move towards earlier treatment lines. Multi-year remission is the goal. But there's real risk these therapies get stuck in later lines. TCEs have been a thorn in cell therapy's side across both oncology and AID - easier dosing, better scalability, cheaper pricing, no lymphodepletion requirements and still solid remission rates. Add in the fact that I&I indications are tightly managed by payors who prefer cycling through cost-effective options first, and the commercial path could narrow considerably. Still, Pharma is clearly seeing enough upside in the platform potential to place big bets - a string of readouts in 2026+ will look to validate that conviction. If you're building or evaluating this space and looking for real-time intel to guide BD or investor workflows, get in touch to learn more about Sleuth's offerings. And as always, comment below for a hi-res PDF!
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Alexey Bersenev
Alexey Bersenev@cells_nnm·
Takeda will be ceasing all work on its cell therapy programs... that's it! BigPharma continues to divest CGT assets! It is massive
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Gene Investing w/Anthony 🧬
Gene Investing w/Anthony 🧬@GeneInvesting·
Why Gene Editing is Finally Making a Move 🚀 1️⃣ $LLY buyout of $VERV 2️⃣ David Liu buying $PRME shares 3️⃣ $51 million insider $CRSP buy 4️⃣ FDA sounding BULLISH on cures 5️⃣ Interest rate cuts coming 🤔 6️⃣ ALL got way too cheap 7️⃣ $NTLA updated HAE Ph. 1 data 8️⃣ $PRME CGD data 9️⃣ $BEAM SCD/AATD data 🔟 Shorts got too comfortable
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Alexey Bersenev@cells_nnm·
@BLLPHD Congrats, Bruce! Amazingly well done! It was great to see you on a bike and on a mountains! And special thinks to @FinckAmanda
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Bruce Levine, Ph.D. 🇺🇦🥼🔬🧬🧪💉
When gifted narrative filmmaker Marwin Gansauge asks, "Can I make a movie about you?", what do you say? In these times we live in, more science communication is desperately needed to convey the importance of research to the development of new treatments for patients. Marwin interviewed me at conferences and meetings in Vancouver and Dublin. youtu.be/YrevpY-DNmw
YouTube video
YouTube
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carolyn johnson
carolyn johnson@Carolynyjohnson·
In case you needed a smile this Tuesday: 10-month-old baby KJ, who received a bespoke gene-editing therapy after a six month scientific sprint - "graduated" from the hospital today and went HOME! YAY SCIENCE! Here's his story: wapo.st/451ZQjT
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Stefanie Bailey
Stefanie Bailey@StefanieBailey_·
Teaching this crew to make virus in the ultracentrifuge today after they successfully cloned and generated their own CAR plasmids 🧬🦠 Super proud mentor 🥹
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Alexey Bersenev@cells_nnm·
The co-founders of the Stem Cell Institute of America and related companies have been banned from offering stem cell therapy and ordered to pay $5 million to consumers who were tricked into buying their unproven therapy consumeraffairs.com/news/stem-cell…
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Patrick J. Hanley 🧬
Patrick J. Hanley 🧬@DrPHanley·
They finally did it! @Mesoblast’s Ryoncil approved for steroid refractory GVHD post stem cell transplant. How well will it be adopted given how much transplant has changed since MSCs first showed potential benefit many years ago? fda.gov/news-events/pr…
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Emily Mullin
Emily Mullin@emilylmullin·
2024 was the year that pig kidneys were transplanted into living people for the first time. March - Richard Slayman, 62, is the 1st to get a pig kidney at Mass General: wired.com/story/pig-kidn…
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Wei Zhao
Wei Zhao@zhaoweiasu·
A must-read for enthusiasts of base editing and fans of $BEAM Therapeutics. Branden Baptiste became the first person in the world to receive base editing therapy (BEAM-101) for sickle cell disease. Suffering from severe and debilitating symptoms, particularly acute chest syndrome (ACS), Branden decided to "take the gamble." He received the treatment on December 5, 2023, and returned home on Christmas Eve—well ahead of schedule. Since then, Branden has described feeling "perfect." answers.childrenshospital.org/sickle-cell-ba…
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Allison Betof, MD, PhD
Allison Betof, MD, PhD@DrBetofMDPhD·
Always an inspiration to hear Dr Stephanie Goff present on #TILs. We need to stop thinking about this as palliative therapy! TILs have the potential to CURE- complete responders from @theNCI with 10 year melanoma-specific survival of 96%. Unmodified TILs are just a starting point. Now, how do get MORE of our patients to a CR? #ImmunotherapyBridge24
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Hannah W. Song, Ph.D.
Hannah W. Song, Ph.D.@hannahwsong·
📢Trainees in cell therapy! The ISCT Leadership Development Program has been a fantastic opportunity to join committees, participate in initiatives, and listen in on conversations on the most up to date issues that process developers are facing. Applications due in January!
ISCT@ISCTglobal

Calling all early-stage professionals! Don't miss this opportunity to gain practical experience on an ISCT expert committee and collaborate on real-world projects alongside Key Opinion Leaders from across the globe. Learn more and apply now: buff.ly/3ZgCNyh

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