Mary-Claire King to Receive Pioneer Award at PMWC 2027

Mary-Claire King, PhD

One of the many illustrious speakers at the Precision Medicine World Conference 2027 (PMWC 2027) (January 27-29, in Santa Clara, California) will be Mary-Claire King, PhD, a pioneering human geneticist best known for proving that breast and ovarian cancer can be inherited. She is Professor of Genome Sciences and of Medical Genetics, U. of Washington School of Medicine. Dr. King will be recognized with a PMWC Pioneer Award for her outstanding work over many years. The PMWC Pioneer Awards are given to venerable individuals whose foresight and ground-breaking contributions to precision medicine propelled the movement in earlier years, allowing it to gain momentum to evolve into the standard of care that it is becoming today. In 1990, Dr. King and her team mapped the locus for the BRCA1 gene to chromosome 17. The finding came after roughly 17 years of research against prevailing scientific opinion. [The exact BRCA1 gene was fully cloned and identified by Mark Skolnick’s group at Myriad Genetics in 1994.] BRCA1 identification transformed cancer diagnosis, risk assessment, drug development, and prevention The mutated BRCA1 gene is responsible for a significant proportion of inherited breast and ovarian cancer cases. Earlier, Dr. King’s doctoral work demonstrated through comparative protein analysis that chimpanzees and humans are 99 percent genetically identical. She also pioneered the use of DNA sequencing for human rights investigations, applying it to identify kidnapped children in Argentina and cases of human rights violations on six continents. Her current work spans inherited breast, ovarian, and prostate cancer; schizophrenia; and severe inherited disorders in children. She is a member of the National Academy of Sciences and the National Academy of Medicine.

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Busy Tissues Need Bigger Cleanup Crews to Work at Their Best

Senior author UCSF’s Jose Angel Nicolas Avila, PhD

The body’s heart muscle (for pumping blood), skeletal muscle (for movement and posture), and brown fat (for heat production), each require significant energy and produce much waste. And that waste needs to be managed. Now scientists from  UC San Francisco, Yale University, University Pompeu Fabra, and the Spanish National Center for Cardiovascular Research have uncovered how tissues adapt their clearance capacity to meet their waste-management needs. In a study published September 4, 2026 in Science Immunology, the researchers found that energy-demanding tissues harbor large clean-up crews of immune cells called macrophages, which means “big eater.” The size of the clearance crew scales with the metabolic activity of each tissue — and the amount of trash they produce. The article is titled “Tissue Mitochondrial Activity Dictates the Macrophage Pool Size.”

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Mahzi Therapeutics Announces FDA Rare Pediatric Disease Designation for MZ-1866 Investigational Therapy for Pitt Hopkins Syndrome

MZ1866 Phase 1/2 UNITE Study is now more than 50% enrolled

On August 25, 2026, Mahzi Therapeutics Inc., a clinical-stage biotechnology company developing precision therapies for neurogenetic disorders, announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to MZ-1866, Mahzi’s investigational gene therapy for the treatment of Pitt Hopkins syndrome. The designation was granted by the FDA’s Office of Orphan Products Development and Office of Pediatric Therapeutics. Pitt Hopkins syndrome (PTHS) is a rare genetic disorder characterized by developmental delay, moderate to severe intellectual disability, distinctive facial features, and possible intermittent hyperventilation followed by apnea. Epilepsy (recurrent seizures) often occurs in Pitt Hopkins. It is part of the clinical spectrum of Rett-like syndromes. Pitt-Hopkins syndrome is clinically similar to Angelman syndrome, Rett syndrome, Mowat–Wilson syndrome, and ATR-X syndrome.

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Multivalent mRNA-Exosome Vaccines: Reshaping Epigenetic and Immune Landscapes to Turn “Cold” Tumors “Hot

Depiction of cross-sectioned exosome

A primary challenge in oncology vaccinology is delivering payloads to lymphoid-resident antigen-presenting cells (APCs) without triggering systemic toxicities or rapid hepatic clearance. While synthetic lipid nanoparticles (LNPs) are standard for systemic delivery, they face hepatocyte sequestration driven by blood-borne apolipoprotein E (ApoE) binding, which limits extrahepatic bioavailability. To bypass this constraint, engineered exosomes offer a biological alternative. These cell-derived vesicles feature a native lipid bilayer enriched with cholesterol and sphingomyelin that shields mRNA from ribonuclease degradation. By presenting surface markers like CD47—a “don’t eat me” signal blocking macrophage phagocytosis via SIRPα—exosomes achieve extended circulation half-lives.

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Nobelist Emmanuelle Charpentier to Receive PMWC’s Inaugural J. Craig Venter Visionary Award–From Reading the Code of Life to Rewriting It

2020 Nobelist Emmanuelle Charpentier

Below is note from Tal Behar, Co-Founder & President of the Precision Medicine World Conference (PMWC), on the PMWC’s new award recognizing the massive contributions and far-reaching vision of genetic scientist J. Craig Venter. Few scientists have changed the trajectory of biology as boldly as he. From helping bring the first draft human genome into view to advancing synthetic biology through the first self-replicating synthetic bacterial cell, his work pushed genomics from discovery into a new era of biological design.

“At PMWC 2027 (January 27-29), we are proud to launch the J. Craig Venter Visionary Award, created to recognize scientists whose work embodies that same spirit of bold, field-defining innovation.”

“We are honored to announce Nobel Laureate Emmanuelle Charpentier as the inaugural recipient. The award will be presented by Heather Kowalski, Acting Chief Executive Officer at the J. Craig Venter Institute.”

“Dr. Charpentier’s co-development of CRISPR-Cas9 genome editing transformed bacterial immune biology into one of the most powerful tools in modern science. Her work opened new possibilities for gene-based therapies, diagnostics, and precision medicine.”

“It is hard to imagine a more fitting first recipient for an award named in honor of J. Craig Venter, a scientist whose career was defined by challenging the limits of what biology can become.”

“Join us at PMWC 2027 as we celebrate this landmark award presentation and the next era of genome engineering.”

Meeting Registration: https://pmwcintl.com/tickets/

–Tal Behar, Co-Founder & President, PMWC

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Four Nobel Laureates Highlight Daunting Cast of 200+ World-Class Speakers at 2027 Precision Medicine World Conference January 27-29 in Silicon Valley

The 2027 Precision Medicine World Conference (PMWC 2027) will feature four Nobel Prize winners among its 200+ world-level speakers at its three-day meeting January 27-29 in Santa Clara, California, the heart of Silicon Valley. Called the “Davos of Precision Medicine,” PMWC27 will include presentations by Nobelists Emmanuelle Charpentier, David Baker, Randy Schekman, and Thomas Südhof. These preeminent scientists will be joined by over 200 notable speakers from across the broad spectrum of precision medicine. See more details below.

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2027 Precision Medicine World Conference (PMWC) January 27-29 in Silicon Valley

The 2027 Precision Medicine World Conference (PMWC 2027) will be held January 27-29 in Santa Clara, California, the heart of Silicon Valley. This premier conference, co-hosted by Stanford, UCSF, and Yale, has been termed the “Davos of Precision Medicine” and every year is amazingly better than the impressive year before. This year’s meeting will feature four Nobel Prize winners and numerous world-class speakers presenting cutting-edge topics in precision medicine. The Nobelists include Emmanuelle Charpentier, David Baker, Randy Schekman, and Thomas Südhof. Among the hundreds of other prominent speakers are Eric Lander, Mary-Claire King, Fei-Fei Li, Sarah Teichmann, Eric Horvitz, Daniel Drucker, Wendy Chung, Andrew Ng, Victor Velculescu, Fei Chen, Christina Curtis, Nir Barzilai, Charles Chiu, Christine Eng, Joseph DeRisi, George Church, Lee Hood, and Catriona Jamieson. “PMWC has proven, time and time again, that it attracts thought leaders from all the relevant fields and catalyzes crucial collaboration through inspiring and practical program content,” commented Lee Hood. You may register to attend this can’t miss conference here.

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2026 Annual Meeting (AAEV) on Extracellular Vesicles November 12-15 in NYC Area

The American Association of Extracellular Vesicles (AAEV) will hold its 2026 annual meeting November 12-15 at the Westin Jersey City Newport in Jersey City, New Jersey, just across the Hudson River from New York City. The meeting will feature numerous cutting-edge presentations by leading EV researchers from around the globe. You may register for this conference here. The abstract submission deadline is September 15, 2026. That is also the deadline for early-bird registration.

Plenary–David Lyden

The opening plenary lecture will be delivered by David Lyden, MD, PhD, Weill Cornell Medical College. Lyden defined the concept of the “pre-metastatic niche” (PMN), where tumor-secreted factors recruit bone marrow-derived progenitor cells to distant organ sites to provide a platform for metastasis. Lyden’s work underscores the systemic nature of cancer. As such, Lyden demonstrated that tumor-derived extracellular vesicle (EV)-mediated crosstalk initiates the PMN and coordinates metastatic progression. He identified key proteins and nucleic acids, specifically double-stranded DNA, in EVs and demonstrated their systemic role in thrombosis, fatty liver metabolic dysfunction, and immune dysregulation. He has defined tumor EV integrins in organotropic metastasis, answering in part Stephen Paget’s “seed and soil” hypothesis on organ-specific metastasis. Moreover, he identified a new particle named “exomere,” which packages distinct proteins, including metabolic enzymes, lipids, and glycans. He has identified novel EV biomarkers for cancer diagnosis and prognosis in patients with cancer.

Two keynote speakers will be Hailing Jin, PhD, University of California, Riverside, and Michel Sadelain, MD, PhD, Columbia University.

Keynote–Hailing Jin

Jin’s research focuses on the roles of RNAs, epigenetic regulation, and antimicrobial peptides in plant–microbe interactions, with the goal of developing innovative and environmentally sustainable strategies for crop protection against fungal and bacterial pathogens. Her laboratory discovered that RNAs, including small RNAs, mRNAs, and long non-coding RNAs, can traffic bidirectionally between plants and their pathogens to regulate gene expression or cellular processes across organismal boundaries. Subsequent work revealed that EVs are key vehicles mediating this cross-kingdom RNA communication.

More recently, her group discovered EV-mediated RNA trafficking between bacteria and fungi, further expanding this biological paradigm. Cross-kingdom/cross-species RNA communication has since been observed across all kingdoms of life, fundamentally changing our understanding of inter-species or inter-organismal communication. Jin’s laboratory also demonstrated that many fungal pathogens can efficiently take up environmental RNAs and vesicles, providing the mechanistic foundation for the development of spray-induced gene silencing (SIGS) technologies for sustainable crop disease control.

In addition, her group has identified several novel classes of plant-derived antimicrobial peptides with broad-spectrum activities that can be harnessed as environmentally friendly biocontrol agents. Jin is a Distinguished Professor and the Cy Mouradick Endowed Chair in the Department of Microbiology & Plant Pathology at the University of California, Riverside.

Keynote–Michel Sadelain

Sadelain is the founding director of the Columbia Institute for Cell Engineering and Therapy at Columbia University, where he is Herbert and Florence Irving Professor of Medicine. He previously founded and directed the Center for Cell Engineering at Memorial Sloan Kettering Cancer Center, where he held the Stephen and Barbara Friedman Chair. He received his MD from the University of Paris, his PhD from the University of Alberta, and trained as a post-doctoral fellow at the Whitehead Institute at MIT. 

Sadelain has made numerous key contributions to the emergence and success of CD19 chimeric antigen receptor (CAR) therapy. His research contributed to all its facets from concept to implementation, including T cell engineering technologies, the design of CARs, the identification of CD19 as an effective CAR target, T cell manufacturing processes, and the clinical translation of CD19 CAR therapy for the treatment of acute lymphoblastic leukemia. 

Other Speakers

Among the other (20+) confirmed speakers are Andrew Hill, Victoria University; Alissa Weaver, Vanderbilt University; Maureen Barr, Rutgers University; Saumya Das, Harvard University; Quanyin Hu, UW-Madison; Huiping Liu, Northwestern; Sathish Ramakrishnan, Yale University; Kenneth Dawson, University College Dublin; and Louise Laurent, UC-San Diego.  

The co-chairs of the AAEV 2026 annual meeting are Ke Cheng, PhD, and Susmita Sahoo, PhD.

Deadlines

The abstract submission deadline is September 15, 2026. That is also the deadline for early-bird registration.

[AAEV 2026 Annual Meeting]

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How Can Stressed Mitochondria Actually Protect Your Heart?

Salk Institute researchers find that placing mitochondria under stress during development protects the adult heart from chemotherapy toxicity in mice

Mitochonrion

Mitochondria are popularly known as the “powerhouse of the cell,” but these cellular structures do so much more—including acting as signaling hubs. Some signals that mitochondria send can reprogram the activity of the entire cell by switching “on” or “off” different genes through epigeneticchanges. Mitochondria’s signals can be so effective that, in yeast, fruit flies, and worms, mild stress in mitochondria early in life can make the entire organism more resilient and live longer through a process called mitohormesis. A new Salk Institute study asks how mitohormesis works in cells and in mice. The authors found that inducing mitochondrial reactive oxygen species (ROS)—byproducts of mitochondria’s energetic function—only during mouse embryonic development is cardioprotective. Then they uncovered the molecular mechanism behind this lasting effect: stressed mitochondria release citrate, initiating a cascade of downstream events that lead to long-term epigenetic changes promoting beneficial mitohormetic adaptations.

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10x Genomics and Lausanne University Hospital Collaborate to Advance Research in Diagnostic Applications of Single-Cell and Spatial Technologies for Cancer Care

On July 29, 2026, 10x Genomics, Inc. (Nasdaq: TXG), the life science technology leader focused on accelerating science and advancing human health, announced a research collaboration with Lausanne University Hospital (CHUV), one of Switzerland’s leading academic medical centers, to advance research in diagnostic applications of single-cell and spatial technologies for cancer care. This collaboration builds on 10x’s broader efforts to work with leading research institutions to generate the scientific evidence needed to advance future diagnostic applications of single-cell and spatial technologies. Through this multi-year collaboration, the study intends to use 10x’s Flex Apex and Xenium platforms, with the goal of expanding to the Atera platform, to examine tumor samples from patients with advanced cancer undergoing a comprehensive evaluation by a clinical molecular tumor board. The research aims to identify clinically relevant biomarkers that may help predict treatment response, cancer prognosis, and support diagnostic development.

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