It is currently only a preprint on bioRxiv, but it has already attracted significant attention from the scientific community and the journal Science. Mammoth Biosciences, a company founded by CRISPR co-inventor Jennifer Doudna, has developed NanoCas, a mini-editor that is just one-third the size of traditional gene-editing scissors (Cas9).
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Have you read Jennifer Doudna’s article in Wired? She discusses how the combination of CRISPR and artificial intelligence could be transformational. In her lab, researchers recently used AI tools to help find “small gene-editing proteins that had been sitting undiscovered in public genome databases because we simply didn’t have the ability to crunch all of the data that we’ve collected”.
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Experimental patients often find themselves in a paradoxical situation: they must be sick enough to qualify for a clinical trial but healthy enough to endure its side effects. They also need the audacity to subject their bodies to protocols whose safety and efficacy remain unproven. For this reason, many describe them as pioneers or even warriors.
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It’s been about a year since the first CRISPR-based treatment was approved in the United States and Europe. However, those expecting a surge in approvals of new gene-editing therapies may be disappointed. Next in line will likely be another approach to treating sickle cell disease, followed by therapies for TTR amyloidosis and hereditary angioedema around 2026-27. According to The CRISPR Journal, that’s all we can expect over the next 3-5 years. Is Casgevy destined to stand out like a cathedral in the desert? We have a super-versatile platform capable of fixing a myriad of genetic defects, so why is the CRISPR revolution slowing down? To understand the looming crisis and the countermeasures needed, don’t miss Fyodor Urnov’s in-depth editorial entitled “Give Cas a Chance: An Actionable Path to a Platform for CRISPR Cures.”
This photo shows the first American “non-experimental” patient leaving the hospital after completing the CRISPR-based treatment for sickle cell anemia (Casgevy). The New York Times detailed this “official first,” which followed the success of a clinical trial involving dozens of patients like Victoria Gray. We still know little about the first person who is beginning treatment in Europe since this therapy became an “approved drug”. According to Osservatorio Terapie Avanzate he is a young adult (23 years), who arrived in Italy in 2014 and living in the Umbria region, where is being also treated. Undergoing cell extraction and reinfusion of edited cells is an invasive and exhausting process, but now the American Kendric Cromer (12 years old) and other “first patients” can hope to lead full lives—without painful crises or blood transfusions. Best of luck!
MicroRNAs won Victor Ambros and Gary Ruvkun the 2024 Nobel Prize in Medicine. Thomas Cech (Nobel Laureate for the discovery of catalytic RNA) has found a fun way to explain how they work. His book, which I reviewed a few weeks ago, is a mine of insights and information. Here is a small excerpt.
Continue readingSammy Basso, 28, the longest-living person with progeria and a brilliant mind, has passed away. He was a biologist passionate about genome editing, and we’d like to remember him through this dialogue with David Liu.
Uditi Saraf died before receiving treatment, but efforts launched for her could help spell a happy ending for other patients awaiting advanced life-saving therapies
Familial encephalopathy with neuroserpin inclusion bodies is a rare neurodegenerative disease with no cure due to the accumulation of toxic proteins in the brain. Depending on the specific mutation, the age of onset can vary greatly. In Uditi Saraf’s case, the first symptoms started early, at age 9. As she worsened, her parents decided to have her genome sequenced, identifying the genetic defect and diagnosing the condition. Their race against time to try to save their daughter was chronicled in an article in Nature, which also offers a glimpse into India’s efforts to make genomic treatments more accessible (see also Nature Biotechnology on gene and cell therapies in the Global South).
After the stunning commercial success of semaglutide-based obesity drugs, the race is on in the biotech world to find a more durable solution that does not require frequent injections. The idea is to silence selected genes without irreversibly intervening on DNA. Basically, it would not involve genetically fixing the target sequence, but preventing its expression through a phenomenon called RNA interference. As is well known, a classical-type gene, in order to express itself, must be transcribed into RNA and then translated into protein. Blocking the transcript, therefore, cancels its action, as Nobel laureates Craig Mello and Andrew Fire have realized.
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Jacob Peckham, 11, can see much better after receiving an experimental CRISPR-based treatment. The American child, a carrier of a genetic defect that impairs the retina, has had surgery on only one eye and hopes to complete the treatment in the future. However, his wish is unlikely to be granted because the company that developed the treatment (Editas) had to abandon the program due to affordability issues. To give a future to treatments for rare diseases such as this one, insists editing pioneer Fyodor Urnov, it is crucial to build a new model for research, development, and production – that is, to simplify, standardize, integrate, scale up.
CRISPeR FRENZY 