Nebulized LNP-formulated DNAI1 mRNA Therapy to Restore Mucociliary Clearance for the Treatment of Primary Ciliary Dyskinesia
2023 Cilia, Mucus and Mucociliary Interactions GRC
is the foundation for our pipeline of disease-modifying mRNA and gene correction therapeutics for genetically defined diseases for which there are few or no current treatments. The SORT LNPs are used to package and deliver genetic medicine cargoes such as mRNA, siRNA, and gene correction modalities.
When delivered into the blood, first-generation LNPs are primarily taken up by the liver, which limits their utility for broad therapeutic applications. ReCode’s SORT LNPs are engineered with a biochemically distinct fifth lipid to help the body “sort” and direct the LNPs to other targeted organs such as the lung and spleen, with the ability to bypass the liver, if desired.
Engineered with a chemically distinct fifth lipid, ReCode’s SORT LNP platform powers a library of LNPs that are tuned to reach specific organs and cells.
Highly selective and predictable organ and cell targeting for the precise delivery of genetic medicines, beyond the liver
A diverse range of cargoes can be engineered for higher quality and potency, including mRNA, siRNA, DNA, gene correction components, and even mixed cargoes
Capability to employ a variety of routes of administration for targeted delivery and biodistribution, including IV, inhaled, subcutaneous, intramuscular, and intrathecal
2023 Cilia, Mucus and Mucociliary Interactions GRC
2023 Cilia, Mucus and Mucociliary Interactions GRC
Ms. Jefferson has nearly 20 years of broad communications and external affairs experience leading public health agencies and biopharma companies.
Most recently, she served as associate commissioner of external affairs for the U.S. Food and Drug Administration (FDA) where she set agency-wide communications strategy, served as the top communications advisor to the commissioner and was the FDA’s principal communications liaison to the White House, the U.S. Department of Health and Human Services and other federal agencies. In this role, she led the primary communications functions for the agency, including media affairs, editorial and creative services, digital and social media and stakeholder engagement.
Before rejoining the agency, Ms. Jefferson was vice president, corporate communications and public affairs at Alector, Inc., a clinical stage neurology company. In this capacity, she built the company’s communications function, led corporate rebranding efforts and supported investor relations. Previously, Ms. Jefferson led communications at Dermira, Inc. (acquired by Eli Lilly), and Pharmacyclics, LLC, an AbbVie company. Early in her career, she held communications roles with increasing responsibility at Genentech Inc. (a member of the Roche Group), the American Pharmacists Association and The McGinn Group (currently McGinn and Company).
In 2021, Ms. Jefferson was named to PR Week’s Health Influencer 30 List. She is a member of the FDA Alumni Association and previously served as a Board Member for the National Association of Continence. Ms. Jefferson holds a master’s degree in health communication from The Johns Hopkins University and a bachelor’s degree in communication from the University of Maryland, College Park.
Rakhshita Dhar is Senior Director of Venture Investments Health at Leaps by Bayer. She joined the team in 2021 and is responsible for search & evaluation and healthcare focused investment for Leaps. She currently sits on the board of Deka Biosciences, Gro Bio, AffiniT therapeutics, Bloom science and Edifice health.
Prior to joining Leaps, Rakhshita was Director of Business Development at Roche Pharmaceuticals where she led collaborations with Dyno Therapeutics and Rheos Biosciences. She also spent a few years at MassBio developing an accelerator program for life-science start-ups.
She got her Undergraduate degree in Biochemistry from Mumbai University and her Masters in Molecular Biology from Georgetown University.
Mr. Hurwitz, an accomplished biotech executive and investor, is a Managing Director at MPM Capital and serves as Chairman of MPM portfolio companies BioIntervene and Rekindle Therapeutics, and on the board of Dyne Therapeutics.
Prior to MPM, he was founder and Managing Director of Precision BioVentures, where he founded and seeded start-up biotechnology companies, including Viewpoint Therapeutics. As a director at Alta Partners, he led 12 successful investments including Applied Genetic Technologies Corporation, Avid Radiopharmaceuticals (acquired by Lilly), Calistoga Pharmaceuticals (acquired by Gilead), Cara Therapeutics, FoldRx Pharmaceuticals (acquired by Pfizer), MacroGenics and Taligen Therapeutics (acquired by Alexion).
Over the course of his career, he has been a Senior Vice President and CFO of Affymetrix, a microarray pioneer, and a biotech research analyst for Robertson Stephens & Company and Smith Barney Shearson. He also practiced law at Cooley Godward LLP.
He earned his J.D. and M.B.A. degrees from the University of California, Berkeley’s Boalt School of Law and Haas School of Business, and his B.A. in molecular biology from Cornell University.
Ms. Kim is a Managing Director of Vida Ventures, a life sciences focused venture capital firm.
Most recently, Ms. Kim was a Partner at The Column Group. Previously, she served as Executive Vice President of Business Development at Kite Pharma, Inc., where she led business and corporate development initiatives including selling Kite Pharma to Gilead in 2017. Previously, Ms. Kim served as Strategic Advisor of NGM Biopharmaceuticals, Inc. from January 2012 through November 2014; served as Chief Business Officer at NGM Biopharmaceuticals, Inc. from August 2009 to January 2012; served as Chief Executive Officer and President of Kosan Biosciences where she restructured and successfully assisted in selling the company to Bristol-Myers Squibb in 2008.
She received a B.S. in chemical engineering from Northwestern University and an M.B.A. from the University of Chicago. Ms. Kim is currently a director for Assembly Biosciences, Applied Molecular Transport, Exicure, Inc., A2 Biotherapeutics, Neogene and PACT.
Mr. Nodelman is the Founder and Portfolio Manager of EcoR1 Capital LLC, a biotech-focused investment advisory firm established in 2013, which invests in companies at all stages of research and development.
With twenty years of experience in biotech investing, Mr. Nodelman has expertise in all aspects of investment management and deep roots in the biotech and scientific communities. Before founding EcoR1, Mr. Nodelman was a portfolio manager at BVF Partners, one of the first hedge funds dedicated to the biotechnology sector. He currently serves as a Board Member for three publicly traded companies: Prothena (NASDAQ: PRTA), a clinical-stage neuroscience company, AnaptysBio (NASDAQ: ANAB), a clinical-stage biotechnology company focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, and Nuvation Bio (NYSE: NUVB), a clinical-stage biopharmaceutical company tackling unmet needs in oncology.
Mr. Nodelman has a Bachelor of Science in Foreign Service with a concentration in Science and Technology from Georgetown University.
Mr. Session II is the President, CEO and founder of Taysha Gene Therapies. Mr. Session has nearly 20 years of experience in the life sciences industry, primarily in business development, corporate strategy and financial roles. Previously, he served as Chief Business Officer of the gene therapy subsidiaries of BridgeBio, as well as Senior Vice President of Corporate Strategy and Business Development at AveXis.
Dr. Lockhart is an accomplished biotech executive with more than 25 years of experience across all phases of drug discovery, drug development and technology development.
Prior to becoming president and CSO of ReCode Therapeutics in January 2022, he served as CEO and president of the company since March 2020. Prior to serving as CEO and president of ReCode, he was CEO and president of its predecessor, TranscripTx from 2014 – 2020. Prior to TranscripTx, he was the Chief Scientific Officer at Amicus Therapeutics from 2006 through 2013. He led a team at Amicus that brought multiple rare disease programs into clinical trials.
Previously, he co-founded Ambit Biosciences, serving as Chief Scientific Officer and president for 5 years, during which the company developed a novel kinase profiling technology and new kinase inhibitors for cancer and other diseases. Prior to Ambit, Dr. Lockhart served as Director of Genomics at the Genomics Institute of the Novartis Research Foundation (GNF). Dr. Lockhart began his biotech career at Affymetrix, where he was the primary inventor and developer of gene expression profiling with DNA microarrays.
Dr. Lockhart received his Ph.D. in Chemistry from Stanford University and was a postdoctoral fellow at the Whitehead Institute in the Biology Department at MIT. He has published more than 80 peer-reviewed scientific articles on pharmacological chaperones, mutation-specific selection of patients for clinical trials, clinical studies with pharmacological chaperones, genomics, kinase technology, kinase inhibitor discovery, and the use of genomic tools to address important biological and biomedical problems. He is also the inventor on more than 40 issued U.S. patents.
Dr. Suliman is a physician, drug developer and dealmaker with over 25 years of experience building and transforming small and large biopharmaceutical companies.
Prior to becoming CEO of ReCode Therapeutics in January 2022, she served as president and chief operating officer of Alector, a leading immuno-neurology and immuno-oncology company where she played a critical role growing, scaling, raising capital and advancing a late-stage development pipeline.
Prior to Alector, she served as senior vice president, corporate development and strategy at Theravance Biopharma from 2017 to 2019, where she helped deliver a $1B profit sharing partnership with Janssen for the company’s lead JAK inhibitor program.
Prior to Theravance, Dr. Suliman was vice president and global head, immunology, infectious diseases and specialty care at Roche from 2015 to 2017. Dr. Suliman worked for Genentech as a group leader and project team leader in the R&D Portfolio Management and Operations Group from 2010 to 2015, where she oversaw an early development portfolio of more than 30 programs across multiple therapeutic areas. She held various management roles of increasing responsibility at Gilead Sciences, Inc. between 2005 and 2010 and played a significant role in forward-integrating Gilead into new therapeutic areas through M&A. Prior to Gilead, Dr. Suliman was an investment banker with Lehman Brothers and Petkevich & Partners, advising numerous public and private companies on buy- and sell-side transactions.
She was named one of the 2017 Fiercest Women in Life Sciences and as one of the National Diversity Council’s Power 50 in 2021 (Most Influential Women in Business). Dr. Suliman serves as an independent director on the Board of Directors of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and 10x Genomics (NASDAQ: TXG). Dr. Suliman received her M.D. (MB, ChB) from the University of Cape Town Medical School, South Africa, and holds an MBA, with distinction, and M.Phil. degrees from Oxford University, where she was a Rhodes Scholar.
Dr. Johnson is an Emeritus Regents’ Professor, Distinguished Professor of Molecular and Cellular Medicine and of Chemistry, and Wehner-Welch Chair at Texas A&M University. He co-founded ReCode Therapeutics and founded tRNA Probes, LLC. His primary research focus has been the production, modification and utilization of transfer RNA (tRNA) molecules for the past 50 years. He was the first to incorporate probes into nascent proteins during their synthesis, using chemically modified aminoacyl-tRNAs to examine protein synthesis, folding, and trafficking, both their insertion into membranes and translocation through membranes. He is responsible for a number of significant discoveries in these and other areas (e.g., pore-forming proteins, blood coagulation) based on the unique research techniques he originated.
He received a B.S. in Chemistry at the California Institute of Technology and a Ph.D. in Molecular Biology/Chemistry at the University of Oregon. After a postdoc in Biophysical Chemistry at Columbia University, he joined the Chemistry and Biochemistry faculty at the University of Oklahoma before moving to Texas A&M University in 1994. He has authored or co-authored over 170 full-length research publications, with an h-index of 75 and more than 17,200 citations.
Dr. Daniel J. Siegwart holds the W. Ray Wallace Distinguished Chair in Molecular Oncology Research at the University of Texas Southwestern Medical Center and is a co-founder of ReCode Therapeutics. He is an Associate Professor in the Department of Biochemistry, Department of Biomedical Engineering (BME), and Simmons Comprehensive Cancer Center (SCCC). He serves as the Director of the Program in Genetic Drug Engineering, Director of the Drug Delivery Program in BME, and Co-leader of the Chemistry and Cancer Program in the SCCC.
His research laboratory utilizes materials chemistry to enable targeted delivery of genomic medicines. He recognized that although great advances had been made in the delivery of short RNAs, the ideal chemical and formulation composition was poorly understood for longer RNA cargo such as messenger RNA. Their efforts led to an understanding of the essential physical and chemical properties of synthetic carriers required for therapeutic delivery of genomic medicines including siRNA, miRNA, tRNA, pDNA, and mRNA.
He has been at the forefront in the design of synthetic carriers for gene editing and has applied these technologies for correction of genetic diseases. The Siegwart lab reported the first non-viral system for in vivo CRISPR/Cas gene editing in December 2016. Recently, they developed Selective ORgan Targeting (SORT), which is the first and only strategy for predictable tissue specific mRNA delivery and gene editing. SORT lipid nanoparticles are systematically engineered to exclusively edit extrahepatic tissues and therapeutically relevant cell types. SORT is compatible with multiple gene editing techniques in targeted tissues.
He received a B.S. in Biochemistry from Lehigh University and a Ph.D. in Chemistry from Carnegie Mellon University with University Professor Krzysztof Matyjaszewski. He also studied as a Research Fellow at the University of Tokyo with Professor Kazunori Kataoka. He then completed a Postdoctoral Fellowship at MIT with Institute Professor Robert Langer and Professor Daniel G. Anderson. Dr. Siegwart has co-authored over 70 research publications, with an h-index of 39 and more than 7,800 citations.
Dr. Thomas is the Ruth S. Harrell Professor of Medical Research in the Department of Physiology at the University of Texas Southwestern Medical Center. Since his arrival in Dallas in 1993, after a postdoctoral fellowship at Johns Hopkins University School of Medicine, he and his group have built on a thirty-five year-long interest in the molecular basis of disease and the translation of this knowledge to patients. This includes contributions to the understanding of genetic diseases owing to protein misfolding and basic knowledge of quality control systems relevant to these pathologies. At Hopkins, he initiated projects into elucidating the molecular pathology of cystic fibrosis, under the umbrella of the CF center. This work provided one of the first examples of protein misfolding as a basis of human disease, along with the suggestion that small molecule ligands might be used to counteract the effects of the most common CF-causing mutation, F508del. Dr Thomas was awarded the JHU Young Investigators award in 1992 for this early work. At UT Southwestern, Thomas and his collaborators defined the details of F508del misfolding and discovered the two-step mechanism that has supported the recent development of effective small molecule therapies that correct the F508del mutant CFTR and restore function. This work was previously recognized by an NIH MERIT award and more recently by the Paul di Sant’Agnese Distinguished Scientific Achievement Award. More recently, the group has led an effort to extend this work to rarer CF causing mutations and their responses to extant drugs, thereby providing “theratype” information for scores of genotypes composed of insufficient numbers to support clinical development. Many of Dr Thomas’ former trainees remain active in CF either as faculty or in commercial efforts to develop better treatments for CF. In addition to his position at UT Southwestern, Dr. Thomas is the senior Principal Scientist at the CFF Laboratory in Lexington Massachusetts and a co-founder of Reata Pharmaceuticals and ReCode Therapeutics, two companies actively translating basic work into new therapies.
Alan Colowick is Senior Managing Director on the Technology-Life Sciences team at Matrix Capital Management Company, LP and its affiliate, AyurMaya Capital Management Company, LP. He currently also serves on the BODs of Acelyrin Inc, Alumis Therapeutics, Solve Therapeutics, Personalis, Inc., AC Immune SA, Harpoon Therapeutics, Inc., InCarda Therapeutics, Inc., XyloCor Therapeutics, and Teon Therapeutics, Inc. Prior to joining Matrix, Dr. Colowick was a Partner at Sofinnova Investments, where he was Chairman of the BOD of two portfolio companies and helped lead their successful acquisitions (Velos Bio acquired by Merck for $2.75B and Principia Inc acquired by Sanofi for $3.7B).
Prior to that, Dr. Colowick had an 18 year career in biotech including significant operating and executive roles at Amgen; Gloucester Pharmaceuticals, where he was CEO and led its acquisition by Celgene: and Celgene, where he was President of EMEA. Dr. Colowick received his MD from Stanford University and completed fellowship training in Hematology and Oncology at the Dana Farber Cancer Institute. He also holds an MPH from Harvard University.
Chris Pritchard is a Principal on the Technology-Life Sciences team at Matrix Capital Management Company, LP and its affiliate, AyurMaya Capital Management Company, LP.
Prior to joining Matrix, Dr. Pritchard was a Vice President at Sana Biotechnology, where he was responsible for business development and strategy leading to numerous successful partnerships, including with Beam and Fujifilm Cellular Dynamics for access to foundational gene editing and stem cell technologies. He also served as a program team leader advancing two novel in vivo gene therapy programs in preclinical research and development.
Prior to Sana, Dr. Pritchard held business development roles at Genentech, resulting in successful acquisitions and clinical stage in-licensing partnerships across therapeutic areas. He was also a project team leader at the Boston Consulting Group, advising major biopharma companies on strategy and operations. Dr. Pritchard received his PhD from MIT under Dr. Robert Langer. He also holds an MBA from MIT Sloan and a Masters from the University of Oxford.
Dr. Amado serves as president, head of global oncology research and development at Zai Labs. He most recently served as executive vice president of research and development at Allogene Therapeutics, Inc., and previously, president of research and development at Adaptimmune Therapeutics PLC, after serving as its chief medical officer. He has held several roles of increasing responsibility at GSK, most recently as senior vice president and head of oncology research and development. Dr. Amado has built a career leading the development of a number of breakthrough therapies in hematology and oncology, with a focus on immunotherapies and CAR-T drug discovery and development.
Dr. Barlow was most recently the chief medical officer of ESCAPE Bio, Inc. Prior to joining ESCAPE Bio, she served as chief executive officer of the Parkinson’s Institute and Clinical Center. Dr. Barlow was also formerly acting chief medical officer at Amicus Therapeutics and was a co-founder, chief scientific officer and chief medical officer of BrainCells, Inc. She also served as director of molecular neuroscience at Merck Research Laboratories and was a professor at the Salk Institute. Dr. Barlow is an expert in neuroscience and neurodegeneration, the treatment of rare and neurological diseases and clinical development for new therapies.
Dr. Sigal currently serves as co-chair of the scientific advisory board for Amgen, Inc. and as a senior advisor to New Enterprise Associates, Inc. He is a member of the Board of Directors for Adaptimmune Therapeutics, Vir Biotechnology, Surface Oncology, Tessera Therapeutics and Affinia Therapeutics. He is a former executive vice president of Bristol Myers Squibb where he also served as chief scientific officer and president of research and development from 2004 until 2013. Under his leadership, BMS brought 14 new medicines to market and became a leading immuno-oncology company. In 2012, SCRIP Intelligence named Dr. Sigal the best R&D chief in the pharmaceutical industry.
Ms. Millican has over 20 years of experience in the life science industry including debt and equity financings, mergers and acquisitions, company formation, licensing transactions, co-development/promotional arrangements, and commercial product launches. Currently, Ms. Millican is a strategic advisor to various life science companies and serves on the board of Life Science Cares, San Diego. Most recently Ms. Millican served as the chief financial officer of Metacrine, Inc. where she led finance and investor relations and raised over $250 million in equity and debt financings including an initial public offering. Prior to Metacrine, Ms. Millican served as the senior vice president of finance at Seragon, a private biotechnology company focused on developing selective estrogen receptor degraders (SERDs) targeting hormone-dependent cancers, which was acquired by Genentech in August 2014.
From July 2012 to August 2013, she served as vice president of finance at Aragon, a discovery-stage small molecule company focused on therapeutics for the treatment of hormone-resistant cancers, which was acquired by Johnson & Johnson in August 2013. From September 2009 to July 2012, Ms. Millican served in various senior financial management roles at Zogenix, Inc., a pharmaceutical company developing and commercializing innovative central nervous system therapies for people living with serious and life-threatening rare central nervous system disorders and medical conditions.
At the beginning of her professional career, Ms. Millican spent five years with the public accounting firm, Deloitte LLP. She holds a bachelor’s degree in Accountancy from the University of San Diego and is a certified public accountant in the state of California.
Ms. Loui brings more than 25 years of pharmaceutical and biotech experience, with a proven track record of leading global organizations through regulatory inspections and product commercialization. Most recently, as founder and chief executive officer of Clarity Global, LLC Consulting, Ms. Loui advised emerging life sciences companies on best practices for development through commercial operations, ensuring compliance with health authority guidelines and alignment with enterprise objectives.
Previously, Ms. Loui served as the vice president of quality at Alector, where she led the build and mobilization of GXP Quality systems in preparation for global clinical trials. Prior to Alector, she was the senior director of CMC clinical quality operations at Gilead Sciences, where she provided leadership for quality CMC strategies across clinical programs. She was also previously the head of quality at Avid Bioservices, leading the organization through multiple successful regulatory inspections and first product approvals.
Ms. Loui spent more than a decade at Genentech, a subsidiary of Roche, in leadership roles including management of Genentech’s external manufacturing quality operations. She has also held positions of increasing responsibility at Bayer Pharmaceuticals in process development, facility startups and regulatory affairs. Ms. Loui earned her MBA from Saint Mary’s College and her bachelor’s degree in Integrative Biology from the University of California, Berkeley.
Dr. Al-Hallaq is Executive Director and Partner at Pfizer Ventures.
Dr. Al-Hallaq leverages her preclinical, clinical, and business development experience to assess, invest in, and manage equity investments for Pfizer Ventures. She currently has responsibility for Pfizer’s investments in Mitokinin, Inc. (San Francisco, CA), Autifony Therapeutics Ltd. (Stevenage, UK), Blade Therapeutics (South San Francisco, CA), RefleXion Medical (Hayward, CA), Montis (Leuven, Belgium), Biograph55 (San Francisco, CA), Vivet Therapeutics (Paris, France), and Arkuda (Cambridge, MA) among others.
Prior to her current role, Dr. Al-Hallaq was a Transactionalist in Worldwide Business Development at Pfizer where she was responsible for negotiating and transacting licenses, acquisitions, and partnerships across therapeutic areas. Dr. Al-Hallaq joined Pfizer in 2015 as an Early Candidate Clinical Lead where she advised early clinical programs in CNS to ensure alignment with business strategies. Prior to joining Pfizer, she held roles at Allergan (formerly Actavis, formerly Forest Laboratories), first in Clinical Development Psychiatry as scientific and operational lead on Phase 2 and Phase 3 studies investigating novel treatments for Major Depressive Disorder and schizophrenia, and later in Business Development where she assessed and executed on a number of acquisitions and licenses across therapeutic areas. She began her training as a research fellow at the National Institutes of Health (NIH).
Dr. Al-Hallaq graduated Summa Cum Laude and Phi Beta Kappa with a B.A. in Biology from Hamilton College and holds a Ph.D. in Neuroscience from Georgetown University Medical Center.