No words. Only feelings of gratitude to Emily, her family, the Penn Medicine team Stephan Grupp MD PhD Carl June Bruce Levine, Ph.D. 🇺🇦 who saved her life - and created an entire new class of medicines.
And a reminder - Emily almost died. The physicians' skill saved her - now we have 35,000 patients!

Exa-cel, CRISPR-edited autologous CD34+ cells to reactivate HbF in severe sickle cell disease. N=44, 97% free from vaso-occlusive crises at 12 month #bmtsm Markus Y. Mapara 🇺🇦 Stephan Grupp MD PhD Akshay Sharma MD @akshaysharma.bsky.social nejm.org/doi/full/10.10…

It sounds like a boring topic, but air conditioning is more important than you realise.

First: there are 2 billion air con units in the world and they account for 10% of all electricity we use.

Second: it has revolutionised architecture and totally reshaped global politics...

The FDA approval of #Beqvez is another milestone in #hemophilia and #genetherapy .

Like #Hemgenix , this #AAV vector uses the hyperactive FIX-Padua.

I wrote about the widespread adoption of FIX-Padua:
cell.com/molecular-ther…

Also, detailed tweetorial:
x.com/drsamelsonjone…

An international consortium led by Dr. Stephan Grupp published final results of a key clinical trial on gene therapy CASGEVY for sickle cell disease treatment. The findings, published in NEJM, led to FDA approval of CASGEVY™ in December 2023.

More: ms.spr.ly/6011YJEX3.

Editorial: Extending Gene Medicines to All in Need nej.md/3QjCLAS

Of 30 patients with severe #SickleCellDisease who were treated with gene-edited autologous hematopoietic stem and progenitor cells, 29 were free from vaso-occlusive crises for at least 12 consecutive months. Read the full CLIMB SCD-121 study results: nej.md/3UvzPDT

Of note, this paper is the result of a truly collaborative, multi-lab effort. Special thanks to @satpathology @joefraietta @gruppsteve Kai Tan 🇺🇦 for enabling this work and helping increase its impact. 17/

.American Cancer Society Professor Anil Rustgi isn't just an outstanding scientist and the Director of Herbert Irving Comprehensive Cancer Center, he's an exceptional mentor who is generous with his time and expertise. Congratulations on being honored at #AACR24 !

2012: Emily Whitehead is 1st person to be treated and cured using CAR-T (by Carl June Stephan Grupp MD PhD and Penn Medicine CMC team Bruce Levine, Ph.D. 🇺🇦).
2024: 35,000 patients treated with CAR-T + TCR-T.
No better #yeswecan story in our field.
HUGE motivator / example for us CRISPR cures people.

In 2009, the U.S. had no solar power and virtually no wind power. Then Obama did a stimulus that put $90 billion into clean energy, which was mocked as a huge failure because Solyndra. Today, a quarter of the grid runs on renewables. grist.org/energy/wind-so…

Ahem

If there was ever a time I wanted people to retweet something - A vaccine specialist, Dr. Paul Offit, spells out how RFK Jr. played a role in discouraging vaccination rates in a small population. A measles outbreak followed which resulted in 83 deaths mostly of young children.

We congratulate the recipient of the AACR Award for Lifetime Achievement in Cancer Research, Steven A. Rosenberg, MD, PhD, FAACR.
Dr. Rosenberg will present his Award Lecture at #AACR24 on Saturday, April 6, 3:00-3:45 PM. Learn more: bit.ly/3TDKGdb

Ortho HPI:

Excited to be in San Diego for the 2nd annual CAR T and Bispecific meeting
Excellent speaker lineup
Looking forward to interactive discussion
cc Dr Raje Noopur Raje Dr Neelapu
MD Education Ryan Fitzpatrick
Christopher Flowers bshah

We're thrilled to announce that our researchers are part of two global teams to be awarded up to $25 million each over five years to take on two of cancer's toughest challenges. #CancerGrandChallenges Cancer Grand Challenges

Learn more at:
ms.spr.ly/6010cbSrm.

Dr. Maus showcased her stellar work with Stefanie Bailey in Blood Cancer Discovery finding dispensability of IFNg in #CARTcell response > now being taken to trials: aacrjournals.org/bloodcancerdis… 2/2
#AACRbcd24 #immunotherapy