Welcome back to this edition of the SynBio for Human Health Newsletter!

It’s been another exciting month for advances in medicine and technology. In a world first, scientists have created the first synthetic human embryos from human stem cells and forgoing the need for eggs and sperm. The researchers emphasize that these model embryos—which resemble the earliest stages of human development—are critical for better understanding human genetic diseases. However, the research raises significant ethical and legal questions though it is unclear if these synthetic embryos could develop beyond their current stage.

The community also celebrated advances in base editing, the next-gen gene editing tool co-developed by David Liu. A collaboration between St. Jude Children’s Research Hospital and MIT has shown promise in addressing sickle-cell disease and beta-thalassemia. By comparing different gene editing techniques, researchers found base editing could significantly increase fetal hemoglobin expression, thereby counteracting the deficient adult hemoglobin production that characterizes these diseases.

And, most recently, Scribe Therapeutics announced an impressive partnership with Sanofi worth more than $1.2b biobucks to tackle sickle-cell disease and other genetic diseases. Founded on CRISPR Cas X IP from Jennifer Doudna’s lab at BerkeleyScribe will develop in vivo payloads for Sanofi’s non-viral delivery system. This comes less than a year after the two companies first partnered on ex vivo therapies.

Also, in this newsletter: 

• Introducing the SynBio for Human Health Podcast!

• Insights: Where can biopharma leverage synbio today? 

• News from the Community 

If you are interested in seeing the impact of Synthetic Biology on Human Health firsthand, join us at the San Jose McEnery Convention Center from May 6-9, 2024, for SynBioBeta 2024: The Global Synthetic Biology Conference. Our Super Early Bird pricing, which offers the biggest discount, ends on Monday, July 31st. Secure your spot today!

Introducing the SynBio for Human Health Podcast!

Tune in to our inaugural episode with bit.bio Founder and CEO, Mark Kotter! We’ll explore how early synbio innovations have already transformed human health, what it will take to overcome our current challenges, and what we can expect for the next generation of therapies. Listen now.

Where Can Biopharma Best Leverage Synbio Today?

Earlier this year, I wrote about the J.P. Morgan Conference in San Francisco and what it taught me about the state of synbio in (or out) of biopharma. Based on what I saw there, I was looking forward to what our community had to say at SynBioBeta’s 2023 Global Synthetic Biology Conference, especially because synbio for human health was a new priority theme. 

While at J.P. Morgan, I was inundated with talks and exhibitors leveraging siloed synbio modalities, from cell and gene therapies to nucleic acid technologies. This doesn’t surprise me—the pharma industry is quite siloed, and different divisions run disparate discovery processes. However, at SynBioBeta, our industry’s biggest thought leaders presented several actions we can ultimately take to bring the synbio stack to biopharma. Despite the promise of synbio and that biopharma is beginning to realize it, we still face a huge uphill battle. But it’s a battle we can win if we approach it right.

Read the full article here → 

News from the Community

• Suddenly, it looks like we’re in the golden age of medicine. Next-gen therapies hit the mainstream in this New York Times Magazine article exploring the upcoming impacts of mRNA therapies on a broad range of hard-to-treat diseases. (New York Times)

• Ginkgo Bioworks completes pilot phase in collaboration with pharma giant, Novo Nordisk - the company achieved the first key milestones in their development for Novo Nordisk and will now advance to improving production processes. (Press release)

• Pearl Bio launches from stealth with 24 patents to develop smart biologics. The cornerstone of the company’s technology, genetically recoded organisms, enables the team to develop novel amino acids and a new class of protein therapeutics. (Forbes)

• New nanopore research could enable advanced diagnostics. Researchers from Aarhus University and Groningen University announced a significant milestone in identifying early-stage human diseases in complex fluids like blood without the need for chemical labeling.

• Are lab-grown kidneys on the horizon? Researchers at the University of Southern California (USC) are making advances on creating lab-grown kidneys. The team has taken inspiration from whale kidneys which resemble a bunch of grapes, meaning the team could potentially achieve the function of human kidneys without having to replicate their complex form. 

• Verb Biotics developing next-gen “precision biotics'' for microbiome health. It’s now understood that the gut microbiome plays a key role in a person’s overall physical and mental health. Verb Biotics is leveraging the power of synbio for targeted delivery of key metabolites. Now in a pre-clinical phase, the first product will launch in Q1 2024 and target digestive health. (AgFunder News)

• AI uncovers novel proteins for base editing. Researchers at the Chinese Academy of Sciences’ Institutes of Genetics and Developmental Biology have developed an AI-assisted method for uncovering novel deaminase proteins. These new proteins could have a major impact on base editing and gene therapies. 

• New hope for myasthenia gravis patients with mRNA CAR-T therapy. In a promising breakthrough, scientists have published results of leveraging a variant of CAR-T to combat myasthenia gravis, an autoimmune disorder affecting the nervous system.

Have news or comments you’d like to share? Reach out to us at humanhealth@synbiobeta.com

That’s all from us this month, but we’ll be back next month with new insights, opinions, and spotlights on the breakthroughs of synbio for human health.

Until next time,

Fiona

Fiona Mischel

Director, Human Health Content and Innovation