Yair Einhorn
@yaireinhorn • 20,228 subscribers
Science and Innovation. I’m laser-focused on BioTech, Pharma, Gene Editing & CRISPR. Occasional thoughts on Stocks, Financial Markets and the Global Economy.
Shorts
Videos
🚨🚨WOW! This is absolutely amazing to see that Karl Deisseroth, Peter Hegemann and Georg Nagel have won the Nobel prize in medicine for their optogenetic research! Visual optogenetics is a therapeutic modality that uses Gene Therapy to deliver a light-sensitive protein to cells in the retina. This mechanism of entering light to the eye can then stimulate these cells to produce a visual signal that is sent to the brain and thus “overriding” the retinal cells and to restore vision to vision impaired patients. This mechanism is already in clinical development and the most advanced optogenetic therapy is currently Nanoscope Therapeutics’ MOGENRY (formally known MCO-010) - an Optogenetic Gene Therapy aimed to restore vision in Retinitis Pigmentosa (RP) patients with a severe vision loss which is waiting for an FDA approval! Absolutely amazing!
Yair Einhorn92,880 Aufrufe • vor 23 Tagen
Here is an excellent answer for a question which I’m asked a lot - could there be a viable business model for a “one & done” Gene Therapy treatment and for CRISPR companies such as $PRME, $BEAM, $LLY, $NTLA, $CRSP, $CRBU, $MGX, $LCTX, $SANA and more. During his excellent conversation with Stripe’s co-founder John collision in the “Cheeky Pint” podcast, Dave Ricks - Eli Lilly’s CEO, presented a new and interesting business model - a multi-year licensing model for genetic medicines based on success which may not only be more efficient but also commercially favourable for patients, insurers and for the companies themselves. Dave Ricks’ licensing model is highly interesting and could bridge the gap between innovation, the patient’s well being and a much needed commercial upside for BioTech and Pharma companies which is a fundamental pillar in any drug R&D process. $XBI
Yair Einhorn196,805 Aufrufe • vor 3 Monaten
It is amazing to see the remarkable advancement that has been already achieved in the Gene Editing field - specifically in Base Editing $BEAM and Prime Editing $PRME 🧵👇. It seems that the words of Prof David R. Liu - the inventor of both of these groundbreaking modalities, about their huge potential to change the lives and to cure some of the hardest medical conditions have never been more true. This is a huge step forward which provides hope to so many patients and their families. Godspeed!
Yair Einhorn14,397 Aufrufe • vor 14 Tagen
I would strongly advise every American leader and policymaker to listen carefully to David R. Liu - the most important scientist and innovator of our lifetime and the person who invented both Base editing & Prime editing - the 2 top leading modalities of Gene editing, when he describes in 60 Minutes the grave danger which the American BioTech ecosystem is facing due to insufficient federal funding. America is already losing its technological and innovative edge! $BEAM $PRME
Yair Einhorn173,977 Aufrufe • vor 11 Monaten
To those who wrongly believe that AI is about to obsolete the need for human intelligence in BioTech R&D I strongly advise to listen carefully to Prof. Jennifer Doudna - the co-inventor of CRISPR Cas9 Gene Editing, a Nobel Prize winner and the co-founder of $NTLA, $SCTX and $CRBU. Anthropic’s recent announcement that Claude had successfully designed novel protein binders from scratch and made a “de novo” design for 14 out of 15 chosen proteins is indeed a huge milestone. Having said that I absolutely agree with Prof. Doudna that it is important to remember that although AI is a powerful tool - it is still a tool, while true innovation and groundbreaking ideas only come from real life scientists. There is no substitute for human creativity and innovation!
Yair Einhorn25,147 Aufrufe • vor 2 Monaten
IMO the most important scientific event this week was the publication of David R. Liu’s new Nature article 🧵👇in which he presented a new and promising Gene Editing modality named PERT - Prime Editing-mediated Readthrough of premature termination codons. This new modality - which uses Prime Editing to converted a regular tRNA gene into a highly optimized suppressor tRNA thus enabling permanent readthrough of premature stop codons, could potentially provide a one-time curative treatment for countless nonsense mutations which are responsible for almost one-third of all human genetic diseases! Here is an excellent lecture by Prof. David Liu - one of the most important scientists and innovators of our lifetime and the person who invented both Base editing and Prime editing - the 2 top leading modalities of Gene Editing, in which he throughly explains this huge scientific breakthrough. $PRME $BEAM
Yair Einhorn62,189 Aufrufe • vor 11 Monaten
One of the most accurate descriptions of the unbearable situation - in which many Gene Therapies and CRISPR Gene Editing therapies fail to reach patients only due to economical and regulatory hurdles 🧵👇, can be found in this statement - which was made during the recent FDA's Cell and Gene Therapy roundtable, by Prof. David R. Liu the inventor of Base and Prime Editing and the Co-founder of $BEAM & $PRME. During his excellent presentation Prof. Liu emphasised the many challenges facing the field including the lack of commercial potential in many cases due to the small number of patients and the need to approve each treatment for one of the 10,000 different rare genetic diseases separately. According to Prof Liu if the sufficient support will be provided by the government - both regulatory and financially, the BioTech and Pharma ecosystem would be able to generate personalised and customised genetic treatments for 1,000 patients each year by 2030, but most importantly would be able to unlock and pave the way for many more patients and treatments down the road. It’s time to accelerate and bring hope to millions of patients and their families worldwide!
Yair Einhorn48,383 Aufrufe • vor 8 Monaten
Alyssa is a 13-year old British girl who has #leukemia.This brave girl is the first person to ever receive a #BaseEditing treatment which led to a complete remission of #Cancer 6 months later! #CRISPR & #GeneEditing give hope to so many! David R. Liu @beamtx
Yair Einhorn169,194 Aufrufe • vor 3 Jahren
This interview of Jake Van Naarden $LLY head of corporate business development, by Angelica Peebles - CNBC’s excellent Pharma reporter, has demonstrated once again just how far-sighted Eli Lilly’s strategy really is and how it helps Eli Lilly to strengthen its dominance in the BioTech and Pharma ecosystem. I was especially interested to learn more about the rationale behind Eli Lilly’s staggering deal-making pace and that there is much more to come M&A-wise. Definitely worth your time! $XBI
Yair Einhorn26,451 Aufrufe • vor 4 Monaten
Here is an excellent conversation of Dr. Scott Gottlieb, MD 🇺🇸 - the former FDA commissioner with Squawk Box’s Becky Quick, in which Dr. Gottlieb discuss the ongoing clinical research & the regulatory efforts made by the FDA in order to find cures for more than 10,000 different rare genetic diseases which affects the lives of more the 30 million Americans and of many millions more worldwide. A very interesting part of this discussion is Dr. Gottlieb’s description of the huge progress made over the past few years in the Gene Therapy and Gene Editing field - including the use of Base Gene Editing by $BEAM and $VERV. Dr. Gottlieb also wonderfully explains - in laymen’s terms the difference between the different genetic delivery modalities such as AAVs and LNPs. Excellent conversation which is definitely worth listening to! $XBI
Yair Einhorn32,348 Aufrufe • vor 9 Monaten
On a day in which stock markets around the world are collapsing - let us all take 4 minutes to watch something inspiring that will help us all to remember what BioTech is all about! Here is the amazing Alyssa - a young British teenager who was the first person to ever receive a Base Editing treatment for Leukaemia Cancer which led to a complete remission! 🧵👇Alyssa spoke in honor of Professor David R. Liu - the inventor of Base Editing platform which literally saved her life, when he received the 2025 #BreakthroughPrize in the Life Sciences. BioTech isn’t just about the numbers but actually about changing and saving people’s lives. Let us not forget that!
Yair Einhorn50,116 Aufrufe • vor 1 Jahr
Here is a much needed dose of optimism by David R. Liu about the huge potential of Gene Editing & how it is changing our lives. The remarkable advancement in the Gene editing field & specifically in Base Editing - $BEAM & Prime Editing $PRME is providing hope to so many patients!
Yair Einhorn40,162 Aufrufe • vor 1 Jahr
🚨WOW! The FDA’s new and promising initiative - regulatory flexibilities for Cell and Gene therapies, is a direct result of the recent Cell & Gene Therapy Roundtable 🧵👇 that was hosted 6 months ago by FDA commissioner Dr. Marty Makary and the director of the FDA’s Center for Biologics Evaluation and Research (CBER) Vinay Prasad MD MPH, in which top industry leaders and scientists shared their views about how to accelerate the R&D process and innovation in the 🇺🇸 BioTech and Pharma ecosystem. It is great to see that this important meeting bore fruit and helped not only to shape the future of the American BioTech ecosystem for years to come but also provided huge hope for patients with genetic diseases worldwide!
Yair Einhorn25,260 Aufrufe • vor 9 Monaten
A new power which threatens the dominance of the American BioTech ecosystem is raising fast in the east. CNN's Will Ripley explains how new & innovative Chinese Biotech companies pose a threat in almost the same way that DeepSeek has done to 🇺🇸 AI. The 🇨🇳 dragon is awake! $XBI
Yair Einhorn39,024 Aufrufe • vor 1 Jahr
President Trump’s new administration could have a huge impact on sectors in which deals have been held back by regulators - such as Pharma & BioTech. If Bill Ackman is correct - we could witness an unprecedented increase in M&A deals in these sectors and others. Godspeed! $XBI
Yair Einhorn32,971 Aufrufe • vor 1 Jahr
For those interested to better understand how CRISPR and Gene Editing work - here a short video of Samarth Kulkarni - $CRSP CEO explaining the mechanism of CASGEVY - $VRTX & $CRSP first ever CRISPR Gene Editing FDA approved treatment for Sickle Cell disease and beta thalassemia. $XBI
Yair Einhorn28,493 Aufrufe • vor 1 Jahr