Session Abstract – PMWC 2025 Silicon Valley
The PMWC 2025 Emerging Therapeutics Showcase will provide a 15-30 minute time slot for selected companies and researchers in the CRISPR, Cell and Gene Therapy fields. Major advancements in safer cell- and gene-level editing technologies are bringing us closer toward cures for life-threatening disorders, from cancer to HIV to Huntington’s disease. Cell therapy, in which cellular material such as T cells capable of fighting cancer cells, is injected into a patient, has been demonstrated safe and effective. The popular new CRISPR tool that has been used to edit the genetic code of nearly any organism will have an enormous impact on human health. More than a dozen clinical trials employing CRISPR on human cells are already underway.
- Emerging Therapeutics/Cell Therapy
- Emerging Therapeutics/CVD
- Emerging Therapeutics/Gene Therapy
- Emerging Therapeutics/Immunotherapy
- Emerging Therapeutics/Radiation Therapy
Confirmed Presenting Companies:
Speaker Profile
Ph.D., CEO, Vector Bioscience Cambridge
Biography
David is a Professor of Molecular Engineering at the University of Cambridge, and a leader revolutionizing cancer treatment with innovative drug delivery systems. David leverages over 15 years of research experience to spearhead advancements in precision medicine. His pioneering work with metal-organic frameworks (MOFs) has led to groundbreaking developments in targeted and controlled release of any modalities, significantly improving existing technologies such as LNPs and ADCs. David has been leading Vector's technology, achieving exceptional drug delivery efficiency and stability, showcasing potential in treating hard-to-treat cancers.
Talk
Revolutionizing Cancer Treatment with Advanced Drug Delivery Systems
Vector Bioscience Cambridge leverages advanced porous nanoshuttles to transform cancer therapy. Our platform offers targeted, controlled drug release of multiple modalities, including functional payloads and RNA, enhancing bioavailability in tumor tissues while minimizing side effects. Discover how our versatile delivery system optimizes therapeutic efficacy, paving the way for groundbreaking advancements in hard-to-treat cancers and beyond.
Emerging Therapeutics Showcase:
Vector Bioscience Cambridge
Vector is pioneering the use of advanced porous nanoshuttles to revolutionize cancer treatment, enabling precision medicine with targeted and controlled drug delivery. Our versatile platform significantly enhances therapeutic efficacy while minimizing side effects, addressing critical challenges in treating hard-to-reach and metastatic cancers.
Speaker Profile
Ph.D., CEO & Founder, Portal
Biography
Armon Sharei, PhD, is the currently the founder and CEO of Portal Biotechnologies - a platform company focused on enabling a new generation of cell therapies and analytics. He was previously the Founder and CEO of SQZ Biotechnologies (NYSE: SQZ), a clinical-stage cell therapy company. During his 10 year tenure, the company raised over $300M in equity financing, established a + $1Bn collaboration with Roche, implemented three oncology clinical trials, and IPOed on the NYSE.Dr. Shareis work on cell engineering has been named as one of Scientific Americans Top 10 World Changing Ideas (2014), Fierce Biotechs Fierce 15 (2015), and a World Economic Forum Technology Pioneer (2017). He has authored over 20 peer-reviewed publications and is the inventor on over 20 patent families.
Talk
Novel Cell Engineering Powered by Portal
Portal will discuss their novel technology that has rapidly gained traction among major biopharma and academic institutions. By dramatically simplifying intracellular delivery, their mechanical delivery technology has enabled novel drug discovery and cell therapy development applications across immune cells, stem cells and other cell types - examples include multiplexed engineering of cells with circular RNA.
Emerging Therapeutics Showcase:
Portal Biotechnologies
Portal is a platform company focused on enabling a new generation of cell therapies and analytics. The mechanoporation technology allows multiplexed delivery of cargo, including RNA, CRISPR, impermeable peptides etc., into immune cells and stem cells while maintaining cell health and function.
Speaker Profile
Program, Alliance, and Business Development Manager, Oxford Biotherapeutics
Biography
Matthew joined Oxford Biotherapeutics in 2022 as a program manager. He is dedicated primarily to external innovation, coordinating and delivering target validation data for partnership programs, and business development, developing content and strategies for future partnerships. He currently oversees the ADC development pipeline of the 10 partnered programs in collaboration with Immunogen. He has a Master of Science from UC Berkeley and brings years of experience in early-stage biotech startups. He is passionate about maximizing the quantity and quality of science produced every day.
Talk
OGAP-Verify: An Enhanced Oncology Target Discovery Platform
OBT is a clinical stage oncology company with a pipeline of first-in-class antibody-based therapies. OBT utilizes OGAP-Verify its proprietary proteomic target discovery platform for ADC, CAR-T and bispecific novel target discovery.
Emerging Therapeutics Showcase:
Oxford Biotherapeutics
OBT is a clinical stage oncology company with a pipeline of first-in-class antibody-based therapies. OBT utilizes OGAP-Verify its proprietary proteomic target discovery platform for ADC, CAR-T and bispecific novel target discovery.
Speaker Profile
Ph.D., CEO, BioEclipse Tx
Biography
Dr. Contag has led both public (President and CEO, Xenogen Corp.,NASDAQ:XGEN), and private companies and is currently the CEO of BioEclipseTherapeutics. As CEO of BioEclipse Dr. Contag was named in the top 100 mostintriguing entrepreneurs of 2018 by Goldman Sachs and in 2020 ranked in thetop 10 of the San Francisco Bay Areas Women-Led most Funded Life ScienceCompanies. BioEclipse was also named one of the top five start-up companiesin the world tackling ovarian cancer in 2021 and 2024, and top 70 startups in SanFrancisco in 2021. In 2022, received as Principle Investigator, an $8M CIRMCLIN2 grant funding the BioEclipse phase I clinical trial. Dr. Contag was alsonamed one of the Top 25 Women in Small Business by Fortune magazine. Sheis widely published and has over 40 patents issued as inventor.
Talk
Optimally Designed Cancer Therapeutics Targeting Tumor Microenvironment
CRX100 carries a specific payload that activates the innate immune system andcauses tumor cell lysis thus influencing the tumor microenvironment by a fulsomeadaptive immune response against tumor-specific antigens, while reliablyinducing tumor cell sensitivity to checkpoint inhibitors.
Emerging Therapeutics Showcase:
BioEclipse Therapeutics
BioEclipse is a clinical stage immune-oncology company in phase 1 trials with
excellent outcomes in late stage ovarian cancer patients. BioEclipse CRX100 is
targeting resistant and refractory late stage solid tumors and can be used as a
monotherapy or in combination with pembrolizumab.
Speaker Profile
Ph.D., CEO, MKC Biotherapeutics
Biography
Successful Biotechnology Executive with private public company experiences in the field of Cell Gene Therapies and ATMP products over the past 3+ decades. The most recent 2+ decades as C-level executive with responsibilities for development of therapeutic products and technology platforms from concept through commercialization FDA approvals. Multiple successful start-up experiences including technology, business, licensing, operations fund-raising financing responsibilities. Operational management (including PL) responsibilities with private and public companies including management of pharmaceutical partner relationships in US Asian territories. Inducted into College of Fellows of the American Institute of Medical and Biological Engineers (AIMBE) for contributions to the development of the field of Cell Gene Therapy.
Talk
Cell-based Therapeutic with Clinical Efficacy with Functional Tissue Regeneration
Our initial product portfolio is based on in-licensing IP Technology encompassing robust scalable pooled allogeneic bone-marrow Mesenchymal Stromal Cell (MSC) platform (NatureSciReports 14:12654, June2024). This platform has resulted in commercial marketing of Products for three indications in India. We will describe data (AmJSportsMed 51:2254 July2023) and plans for pivotal trials for knee osteoarthritis in USEurope.
Emerging Therapeutics Showcase:
MKC Biotherapeutics, Inc.
MKC Biotherapeutics business model to identify and in-license novel therapeutic platforms / product assets with demonstrated human safety & efficacy data with durable clinical benefit (ideally, commercially marketed in select countries outside US/Europe under country-specific-FDA approval) and undertake clinical / regulatory development for commercial markets in US/Europe.
Speaker Profile
M.D., Ph.D., Assistant Professor of Medicine, Dana Farber Cancer Institute
Biography
Baochun Zhang is an Assistant Professor of Medicine at the Dana-Farber Cancer Institute and Harvard Medical School. His research has advanced our understanding of the molecular mechanisms underlying B-cell lymphoma development. His group has unveiled that the Epstein-Barr virus, a known tumor virus, can provoke a broad anti-tumor immune response, opening the door to understanding the potential impact of EBV-induced immunity on a wide range of EBV-negative cancers. Dr. Zhangs work has elucidated the formation of cytotoxic CD4+ T cells, particularly focusing on the role of the CD70CD27 and OX40LOX40 costimulatory pathways in this process. Building on these insights, he is pioneering innovative strategies to generate cytotoxic CD4+ T cells targeting multiple antigens for immunotherapy in both liquid and solid cancers. His efforts promise to enhance cancer research and treatment, ultimately leading to improved health outcomes.
Talk
Multispecific Cytotoxic CD4+ T-cells for Cancer Therapy
Recent advances in tumor immunity highlight the potential of CD4+ cytotoxic T-cells (CD4 CTLs) in cancer therapy, offering benefits for long-term anti-tumor immunity and safety. I will present our findings on developing and translating multiantigen-targeted CD4 CTL therapies for liquid and solid cancers.
Emerging Therapeutics Showcase:
Dana-Farber
Since its founding in 1947, Dana-Farber Cancer Institute in Boston, Massachusetts has been committed to providing adults and children with cancer with the best treatment available today while developing tomorrow's cures through cutting-edge research.
Speaker Profile
M.D., Ph.D., Founder & Partner, Genesis BioCapital
Biography
A pioneer in the field of CancerImmunotherapy and next-generationtherapies; co-author of cancerimmunity cycle and the cancer immune set point. Former VP at GenentechRoche and former CMO at IGM Biosciences;Experienced drug developer that led development and global approvals for Tecentriq (anti-PDL1),entrepreneur, executive andstrategic leader;
Talk
Genesis BioCapital: Creating the Future of Medicine
Focus on Innovation, Technology, Biomarkers and Patient Selection and Company Formation
Emerging Therapeutics Showcase:
Genesis BioCapital
Envisioning a new way for company formation and venture growth in transformative medicines
Speaker Profile
Ph.D., Director, Advance Genomics, Resilience
Biography
Neeraj Salathia has over 15 years of experience working in the biopharmaceutical industry and currently directs the Advanced Genomics RD team at Resilience. His work focusses on understanding the biological characteristics of complex medicines, such as cell- and gene- therapies, as they relate to biomanufacturing processes and ultimately clinical efficacy. Prior to joining Resilience, Neeraj led genomics-centric research efforts at Bristol Myers Squibb, supporting drug discovery research covering neurobiology, oncology and autoimmune disease indications. He has also worked at Celgene and Illumina developing novel assays for single cell biology and non-invasive cancer detection. Neeraj trained in molecular genetics and received his graduate degree from the University of Warwick, UK, followed by postdoctoral research at Harvard University and Novartis.
Talk
Rapid Multi-Omics Cell Therapy Characterization and QC
Resilience aims to translate advances in cell therapy research for patient benefit by developing a multi-omics assay platform for characterization of cell therapy products and consolidated QC release. This solution will significantly reduce manufacturing times and costs while enhancing understanding of drug product characteristics and associated patient clinical outcomes.
Emerging Therapeutics Showcase:
Resilience
Resilience is a technology-focused biomanufacturing company driving the pursuit of novel medicines across diverse disease modalities. We harness innovation to revolutionize how these therapeutics are made, funded and scaled.
Speaker Profile
MBA, CEO, EveryONE Medicines
Biography
Kent Rogers has been a senior executive across the healthcare industry for over 30 years, focusing on product commercialization, supply chain logistics, pricing strategies, reimbursement, market access, and alliance management. He spent more than two decades in the pharmaceutical industry in multiple leadership and executive roles at several top-tier pharmaceutical firms as well as biotech startups. Kent also worked on the payer side of the industry at OptumRx, a national PBM for United Health Group. Today, Kent is with EveryONE Medicines, a biotech startup focused on developing medications to treat n-of-1, neurodegenerative diseases. He sits on several boards and currently serves as a Partner with ARCH Venture Partners.
Talk
Treating The Rarest Diseases At Scale
There is no regulatory pathway for approving medicines to treat so-called n-of-1 diseases. New guidance has been issued in the US and there is movement in that direction in the EU. A scalable and sustainable solution is possible now.
Emerging Therapeutics Showcase:
EveryONE Medicines
Speaker Profile
Ph.D., Lead Application Scientist, VectorBuilder
Biography
Dr. Bai provides technical consultation on in vitro and in vivo gene delivery for research and clinical projects. He is experienced in design both viral (AAV, lentivirus, adenovirus) and non-viral (plasmid, in vitro transcribed mRNA, lipid nanoparticles) vectors.
Talk
Basics of IVT mRNA from Design to Therapy
In vitro transcribed (IVT) mRNA emerged as a promising therapeutic compound in the fight against the SARS-CoV-2 pandemic, offering distinct advantages over traditional tools like viral vectors. In this seminar, we will discuss the therapeutic applications of mRNA and producing potent mRNA with optimized design and chemically modified nucleotides.
Emerging Therapeutics Showcase:
VectorBuilder
VectorBuilder is a global leader in gene delivery technologies. As a trusted partner for thousands of labs and biotech/pharma companies across the globe, VectorBuilder offers a full spectrum of gene delivery solutions covering virtually all research and clinical needs from bench to bedside.
Speaker Profile
Other, Director, Pacylex Pharma
Biography
Michael J. Kamdar is an experienced life science and healthcare executive with over 35 years of international leadership experience. He is an industry veteran that has done over $1.5B in business deals and raised more than $750 million from venture firms and public markets. Mr. Kamdar has led andor founded a number of life science and healthcare companies, including Molecular Assemblies (President CEO and Director), Ciclofilin Pharmaceuticals (Founder and Director, which acquired by Hepion), VentiRx Pharmaceuticals (Founder, CEO and Director, which was subsequently acquired by Celgene), Avalyn Pharmaceuticals (Founder, CBO and Director), Anadys Pharmaceuticals (CBO and CFO) (Nasdaq: ANDS) and Agouron Pharmaceuticals (Head of Global Business Development) (Nasdaq: AGPH) which was acquired by Pfizer. He currently sits on the Board of AeenaDx and the Strategic Advisory Board of YatiriBio.
Talk
NMT Inhibitors: New ADC Payloads for Cancer
Cancer hijacks processes involving myristoylation, becoming more dependent on this for survival than normal cells. Phase 1 study of zelenirstat, Pacylexs first in class N-myristoyltransferase inhibitor (NMTi) demonstrated acceptable safety and signs of efficacy in solid tumor patients. NMTi properties are attractive for ADC payloads, especially for solid tumors.
Emerging Therapeutics Showcase:
Pacylex Pharmaceuticals
Pacylex Pharmaceuticals is the world leader in the clinical development of N-myristoyltransferase inhibition (NMTi) therapy for cancer. Pacylex has exclusive license to a family of 503 small molecule NMTis, 28 of which have single nM IC50s against human NMT1.
Speaker Profile
Ph.D., Director, Discovery Research, Neurona Tx
Biography
Marina Bershteyn is one of the founding scientists on the Neurona Therapeutics discovery team. She co-led the development of a novel stem cell-derived inhibitory cell therapy for mesial temporal lobe epilepsy, from early-stage protocol development and product characterization, through the IND approval by the FDA for First-In-Human Phase III Clinical Study. Prior to joining Neurona, Marina did her postdoctoral research in developmental neuroscience at UCSF, where she established the first patient-derived induced pluripotent stem cells (iPSCs) from lissencephaly and ring chromosome disorders and implemented 3D cerebral organoid models together with live imaging and single cell transcriptomics to study the mechanisms of human neurodevelopmental disorders. She obtained her PhD in cancer biology from Stanford University, where she investigated epithelial-mesenchymal Sonic Hedgehog (Shh) signaling and cytoskeletal regulation in the context of skin cancer and hair regeneration.
Talk
Interneuron Cell Therapy for Drug-resistant Focal Epilepsy
Temporal lobe epilepsy is the most common type of adult focal epilepsy, with one-third of individuals experiencing refractory seizures. We developed a functionally restorative cell therapy comprising GABAergic interneurons to replenish the corresponding endogenous population, which is compromised in TLE. The talk will cover preclinical and clinical progress to date.
Emerging Therapeutics Showcase:
Neurona Therapeutics
Neurona Therapeutics is a clinical-stage biotherapeutics company focused on discovering and developing allogeneic neural cell therapies to treat chronic diseases of the nervous system.
Speaker Profile
RPh, CCN, DHPh, Founder of Metabolic Code Enterprises, TrueBinding
Biography
James LaValle is a globally recognized clinical pharmacist, author, and board-certified nutritionist with over 40 years of experience in precision health and integrative care. Founder of Metabolic Code Enterprises, he developed a cloud-based platform to identify metabolic health roadblocks. LaValle served as faculty at the University of Cincinnati College of Pharmacy and the George Washington School of Medicine, among others.He is Clinical Co-Chair at the American Academy of Anti-Aging Medicine and Chair of the International Peptide Society. LaValle has advised elite organizations like the Orlando Magic, Chicago Blackhawks, and Golden State Warriors on performance and recovery. Former Clinical Director for the Pro Football Hall of Fame Performance Health Program, he also educates first responders and military on recovery programs.Author of over 26 books, including Cracking the Metabolic Code, LaValle is Chief Science Officer at Life Time, leading its longevity and performance program, MIORA.
Talk
Galectin-3: Target to Reverse Decline of All Dementias
The presentation, "Galectin-3: The Potential Target to Reverse Decline of All Dementias" by James LaValle, explores the critical role of Galectin-3 in neuroinflammation, its association with chronic diseases, and its impact on dementia progression. It highlights groundbreaking findings on Galectin-3's mechanisms, therapeutic targets, and emerging treatments like TB006, aiming to advance dementia care.
Emerging Therapeutics Showcase:
TrueBinding
The Metabolic Code is a proprietary clinical assessment approach bridging practitioners and patients to address self-care and lifestyle goals. Its supplements target the 5 TRIADS, addressing specific metabolic imbalances identified through personalized reports, fostering health transformation and aspirational living.
Speaker Profile
Ph.D., Chief Scientific Officer, Nature's Toolbox (NTx)
Biography
Dr. Steven Evans is the Chief Scientific Officer at NTx, bringing over 20 years of innovation-driven drug discovery and development experience across multiple therapeutic modalities and disease indications. He has been pivotal in advancing solutions that bridge the gap between cutting-edge research and clinical application. NTx has made significant strides in continuous flow synthesis, leading to the production of next-generation RNA and protein platforms. These advancements offer the speed and scalability necessary to cost-effectively address unmet needs, from global pandemic responses to personalized precision medicines. Dr. Evans has authored numerous peer-reviewed publications and holds several patents in the field.
Talk
Revolutionizing Biomanufacturing: Rapidly Deployable Continuous Flow RNA Manufacturing Platform
Natures Toolbox (NTx) develops innovative biomanufacturing solutions like the NTxscribe platform, a proprietary continuous-flow IVT system using hollow fiber bioreactors. Designed for cost-effective, flexible, and rapid mRNA production, it supports distributed manufacturing models, modernizing RNA production with improved speed, quality, and adaptability to meet growing vaccine and therapeutic demands.
Emerging Therapeutics Showcase:
Nature's Toolbox (NTx)
NTx, based in Rio Rancho, NM, is developing innovative systems like NTxpress® and NTxscribe® to enable manufacturing of mRNA vaccines and protein therapeutics in an eco-friendly and sustainable way. These easily scalable processes enable NTx’s platform to scale from personalized medicine dosage to pandemic volume.
Speaker Profile
Ph.D., CEO, NysnoBio
Biography
Jennifer is the creative force behind NysnoBio, responsible for the business and scientific focus of the company as CEO. She has over 20 years of experience in the biopharmaceutical industry, including 13 years in neurodegenerative diseases at Elan Pharmaceuticals.As VP, Head of Discovery Research at Elan, Jennifer delivered multiple novel molecules for Parkinsons disease from initial discovery to candidate selection.In 2019, Jennifer founded NysnoBio to focus on gene therapy approaches. She has more than ten patents and over 40 publications. She has served as a reviewer for multiple scientific journals, and on the SAB of the Michael J. Fox Foundation for over 18 years.Outside the office, Jennifer has been a member of the USA Track Field 100Km Team, earning bronze and silver medals in the national championships, as well as holding the record for the most wins in the Angeles Crest 100-mile trail race.
Talk
Parkin Gene Therapy for Parkinson's Disease
NysnoBio is advancing a novel gene therapy to restore mitochondrial function in Parkinson's Disease (PD). Loss of the Parkin gene is a genetic cause of PD, and Parkin's role to restore damaged mitochondria demonstrates a unique opportunity to treat genetic and sporadic populations of the second most common neurodegenerative disorder by replacing the Parkin gene.
Emerging Therapeutics Showcase:
NysnoBio
NysnoBio is advancing genetic therapies to treat rare and common neurodegenerative disorders. Our platform is aimed at addressing disease at the root cause with restorative therapies having broad application for therapeutic benefit.
