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Hi ,
Welcome back to this month’s edition of our human health newsletter. There’s been big news for synbio-backed therapeutics as well as significant deals between synbio companies and pharma. As we wrap up 2023 and look forward to 2024, this edition brings you:
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Top 9 SynBio for Human Health Stories We Loved From 2023
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#1 World-First CRISPR-based Therapeutic Approved for Sickle Cell Anemia
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Image via Canva
How could this not be our number one story? When CRISPR Cas9 technology was revealed to the world in 2012, it was hailed as one of—if not the—most significant scientific breakthroughs of the 21st century. CRISPR's “genetic scissors” were set to reshape medicine and the bioeconomy, bringing new therapies to patients and sustainable products to the market. The rarified cherry on top was the honor of the Nobel Prize for the technology’s discoverers. Eleven years have passed since Jennifer Doudna and Emmanuelle Charpentier’s CRISPR seminal paper. Despite steady progress (and explosive patent battles), no approved drugs have arrived for patients. That was, until a few weeks ago.
The long-time partnership between Vertex Pharmaceuticals and CRISPR Therapeutics has finally borne fruit. In a groundbreaking announcement, the Medicines and Healthcare products Regulatory Agency of the UK (MHRA) granted conditional marketing authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR Cas9 gene-edited therapy designed to address sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
Exa-cel is currently under review by the European Medicines Agency, the Saudi Food and Drug Authority, and the U.S. Food and Drug Administration (FDA). The FDA assigned a Priority Review for SCD, which was approved on December 8th. The FDA also set a Standard Review for TDT by March 30, 2024. While the stock price of CRISPR Therapeutics slumped dramatically following the FDA’s first approval, we still expect significant success from the therapy, even if it takes longer than expected to deliver financial returns. Patients’ lives will be saved. That’s CRISPR’s big win.
#2 A Nobel Prize for mRNA Technology
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Katalin Karikó, PhD, and Drew Weissman, MD, PhD. Photo Credit: Peggy Peterson Photography for Penn Medicine.
The mRNA COVID-19 vaccines are widely credited for ending the pandemic years sooner than expected, saving millions of lives. Earlier this year, the pioneers of mRNA vaccine technology were awarded the Nobel Prize in Physiology or Medicine. The Nobel winners, Katalin Karikó and her longtime collaborator, Drew Weissman, faced a twenty-year uphill battle to get the power of their technology recognized. Karikó’s now-famous story is particularly poignant. She moved from lab position to lab position, never gaining her own lab, acclaim, or even grants to continue her work. It’s a familiar tale in the trials of academia, but Karikó was not deterred. The end of the COVID-19 pandemic and the birth of mRNA technology across the drug industry is the payoff of Karikó and Weissman’s persistence.
#3 US Bold Goals Strategy for Cell Therapy
This story comes from early 2023 when the Biden Administration announced its Bold Goals roadmap for biomanufacturing. Many of these goals were centered on the climate crisis, but there were also big announcements for human health. Most notably, one goal is to increase cell therapies by 10x while reducing costs by 10x within the next 20 years. This announcement came alongside the creation of a new US ARPA agency, ARPA-Health. Since then, ARPA-H has founded its three hubs (Cambridge, Massachusetts, Dallas, and Washington, DC) and has launched a range of initiatives and funding challenges.
#4 Absci Wins Big in Big Pharma
AI therapeutic design company Absci has been making noise in the human health space for several years, including a 2022 deal worth up to $610 million with pharma giant Merck. The company continued its big pharma deal streak with a new collaboration with AstraZeneca. The deal, worth up to $247 million, combines Absci’s AI antibody drug creation platform with AstraZeneca’s oncology research and development expertise and aims to deliver an AI-designed antibody against AZ’s oncology target. SynBioBeta CEO John Cumbers met with Absci CEO Sean McClain to discuss these deals and more in SynBioBeta’s latest podcast—be sure to take a listen!
#5 Neurotech Takes Huge Strides
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Image via Canva.
This isn’t one single story but many. This year, Neurotech saw wide-ranging successes, from enabling ALS patients to speak
again to allowing a patient with severe Parkinson’s to go for regular, unaided 6 km walks. I sat down with investor, neurotech expert, and 2024 Conference Track Chair Amy Kruse to better understand the synbio-neurotech connection and discover where the field is heading. Read the full story here!
#6 The AI Race is on in Human Health—Is Europe Taking an Unexpected Lead?
There’s been one topic on everybody’s minds in 2023—ArtificaI Intelligence. AI threats, AI promises, AI, everything in between. Human health is no exception. But it may surprise you—as it surprised me—to learn that American Big Pharma may not be leading the way for once. The US has a reputation as a first adopter (for better or worse), but this may not be the case this time around. I sat down with ML/ AI leader and CEO of Cradle, Stef van Grieken, to discuss the AI opportunity space for human health and to see what big gaps the field may be overlooking. Check out the full insights here!
#7 Phages and Synthetic Biology Team Up to Tackle Antimicrobial Resistance
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Image via Canva.
Synbio is founded on our ability to connect and layer bioengineering technologies. This year, a new pairing hit the
scene in the form of synbio and phage therapy for antimicrobial resistance (AMR). The growing AMR crisis can often be overlooked in the face of more front-of-mind threats such as cancer or tropical diseases. But, the World Health Organization cites AMR as “one of the top public health and development threats.” The WHO’s warning is so dire that it
estimates AMR could undo much of the progress of modern medicine. Not only would humans and animals be vulnerable to simple infections, but the efficacy of surgeries and other life-saving procedures would also become extremely limited. Needless to say, we need a new generation of AMR options that can adapt as pathogens evolve. We’ll be following this story closely in the coming years.
#8 Novel Gene Therapy Offers a Potential Solution for Severe Addiction
Cell and gene therapies have been touted as the answer to a whole host of hard-to-cure conditions, from neurodegenerative diseases to blood disorders. But a surprising use case struck us this year: the potential to use gene therapy to treat severe alcohol use disorder. I spoke with Dr. Krystof Bankiewicz, one of the lead authors of this study, to gain insights into this novel treatment approach as well as to discuss the potential medical and ethical risks associated with using gene therapy to alter a person’s reward system. See the full interview and explore the breakthrough here.
#9 Using Probiotics to Guide CAR-T Cells Through the Tumultuous Terrain of Solid Tumors
Penetrating solid tumors is the holy grail of oncology. Getting past a tumor’s defenses and into the region where a therapeutic can be effective is akin to navigating a minefield. One of the critical obstacles is the tumor microbiome. While therapies like CAR-Ts can act well against blood cancers, they are still very limited when it comes to solid tumors. Earlier this year, a team at the University of Columbia used a non-pathogenic strain of E.coli to guide CAR-T cells through the tumor environment as well as to amplify their attack against the cancer cells. Their proof of concept results were effective, and the CAR-T cells successfully reached and shrank the tumor. We’ll be following this
story closely to see what comes next.
That’s all from us for 2023! We hope human health innovation in 2024 will build on these initial synbio success stories and bring more therapies to the patients who need them most.
News from the Community
Industry News |
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Have news or comments you’d like to share? Reach out to us at humanhealth@synbiobeta.com and subscribe here: https://www.synbiobeta.com/sign-up-for-our-digest
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
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