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ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT2100 for the Treatment of Cystic Fibrosis

Menlo Park, Calif. – March 25, 2025 – ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for RCT2100, a novel, investigational mRNA therapy being developed to treat cystic fibrosis (CF).

CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Although recent advancements in CFTR modulator therapies have improved outcomes for many people with CF, those with certain mutations remain underserved because these mutations prevent the production of functional CFTR protein, making current treatments ineffective.

RCT2100 was formulated leveraging ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform and is designed to deliver CFTR mRNA directly to target cells in the lungs, instructing them to produce a functional version of the CFTR protein that is missing in some individuals with CF. By providing this CFTR mRNA, the therapy aims to address the root cause of CF, potentially restoring CFTR protein function in the lungs.

“While there have been tremendous advancements in the development of novel CF therapies in the past two decades, we are focused on the subset of CF patients who are not eligible for current treatments and who have been waiting for a treatment,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. “We are currently enrolling patients in a Phase 1b study evaluating RCT2100 in people with CF who do not respond to or are intolerant of current modulator therapies. We are grateful to the cystic fibrosis community and physicians for the continued support of the RCT2100 clinical program.”

A Phase 1b study evaluating RCT2100 is currently enrolling individuals with CF in the United States, United Kingdom, France, and the Netherlands. For more information, please visit CF-Clinical-Studies.com.

About Cystic Fibrosis

Cystic fibrosis (CF) is a progressive genetic disease that causes persistent lung infections and respiratory failure. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. An estimated 105,000 people have been diagnosed with CF across 94 countries, according to the Cystic Fibrosis Foundation. The absence or dysfunction of the CFTR protein results in a defect in airway hydration, which leads to excessive mucus buildup in the lungs and other organs. It also results in a mucociliary clearance defect, recurrent infections, inflammation, respiratory failure, and other complications. Despite advancements in CFTR modulator treatments, approximately 10% of people in the CF community have genetic mutations that do not enable them to benefit from these life-changing therapeutics.

About ReCode Therapeutics

ReCode Therapeutics is a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs, tissues, and cells implicated in disease, enabling improved efficacy and potency.

ReCode’s lead programs include RCT2100 for the treatment of the 10% of people with cystic fibrosis who have genetic mutations in the CFTR gene that do not respond to currently approved CFTR modulators or those that are intolerant of approved CFTR modulators and RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene. RCT1100 and RCT2100 are inhaled disease-modifying mRNA-based therapies formulated with the SORT LNP delivery platform. ReCode is expanding its pipeline to develop potential therapies for other rare and common genetic diseases, including central nervous system, liver, and infectious disease indications.

ReCode has been recognized by the San Francisco Business Times and Silicon Valley Business Journal as a Best Place to Work. For more information, visit www.recodetx.com and follow us on LinkedIn and Instagram.

ReCode Contacts

Investors:
Anne Marie Fields
Stern IR
[email protected]
[email protected]

Media:

Erica Jefferson
Senior Vice President, Corporate Affairs
[email protected]

Tara Cooper
The Grace Group
[email protected]

Andrew Bellinger, M.D., Ph.D.

Scientific Advisory Board Member
Dr. Bellinger brings a deep understanding of gene correction, genetic payload discovery and rare disease drug development experience to ReCode’s SAB. Most recently, he served as chief scientific officer at Verve Therapeutics. Just prior, he co-founded and served as chief scientific officer of Lyndra Therapeutics, where he helped to build the company’s research team and was involved in the company’s key external partnerships.

Alan Colowick, M.D., M.P.H.

Matrix Capital Management Company

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.

Ed Hurwitz, J.D., M.B.A.

MPM Capital

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.

Helen Kim, M.B.A.

Vida Ventures

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.

Basheer Zada

EcoR1 Capital
Basheer Zada is a Principal at EcoR1 Capital. Prior to joining EcoR1 in 2018, Mr. Zada was a Senior Associate at Brace Pharma Capital, a life sciences focused venture capital firm. Mr. Zada currently serves as a board member for Actio Biosciences. Mr. Zada holds a Bachelor of Science degree in Biology from Virginia Tech and a Master of Science degree in Biotechnology from Georgetown University.

Peter Thompson, M.D.

OrbiMed Advisors
Dr. Thompson is a partner on the private equity team at OrbiMed Advisors LLC. With over 25 years of industry experience, he has held executive positions at Becton-Dickinson and Chiron, co-founded and was CEO of Trubion Pharmaceuticals, co-founded Corvus Pharmaceuticals, Cleave Biosciences, Silverback Therapeutics and serves as a director on several company boards. Dr. Thompson is an Ernst & Young Entrepreneur of the Year awardee, an Affiliate Professor of Neurosurgery at the University of Washington, an inventor on numerous patents and a board-certified internist and oncologist. He was on staff at the National Cancer Institute following his internal medicine training at Yale University.

Shehnaaz Suliman, M.D., M.B.A., M.Phil.

ReCode Therapeutics

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.

Rakhshita Dhar, M.S.

Leaps by Bayer

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.

Daniel J. Siegwart, Ph.D.

Chair of the Scientific Advisory Board
Dr. Siegwart is the W. Ray Wallace Distinguished Chair in Molecular Oncology Research and associate professor in the Department of Biochemistry at the University of Texas Southwestern Medical Center and is a co-founder of ReCode Therapeutics. Dr. Siegwart has been a pioneer in the design of delivery technologies for genetic medicines. ReCode’s SORT LNP platform, developed by Dr. Siegwart and his lab, was described by Nature as one of the “Seven Technologies to Watch in 2022.” In 2016, his lab reported the first non-viral system for in vivo CRISPR/Cas gene editing. Dr. Siegwart and his research team are focused on solving challenges in disease therapy and diagnosis, with a focus on the development of new materials that can deliver genetic medicines to treat cancer and genetic diseases.

Katalin Karikó, Ph.D.

Scientific Advisory Board Member
Dr. Karikó is a world-renowned biochemist and researcher who was awarded the 2023 Nobel Prize in Medicine alongside Drew Weissman, M.D., Ph.D., for their discoveries that enabled the use of modified mRNA, which was critical for the success of COVID-19 vaccines, and has laid the groundwork for the development of mRNA therapeutics. Dr. Karikó also has a long-standing interest in developing therapies for cystic fibrosis and other genetic diseases.

Rafael Amado, M.D.

Scientific Advisory Board Member

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.

Carrolee Barlow, M.D., Ph.D.

Scientific Advisory Board Member

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.

Eric Olson, Ph.D.

Scientific Advisory Board Member
Dr. Olson is the founding chair of the department of molecular biology at the University of Texas Southwestern Medical Center. He also directs the Hamon Center for Regenerative Science and Medicine and the Wellstone Center for Muscular Dystrophy Research. He holds the Robert A. Welch Distinguished Chair, the Pogue Distinguished Chair and the Annie and Willie Nelson Professorship. Dr. Olson and his team discovered many of the genes that control heart and muscle development and disease. His most recent work has provided a new strategy for correction of Duchenne muscular dystrophy-causing mutations using CRISPR gene editing.

Elliott Sigal, M.D., Ph.D.

Scientific Advisor

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.

Justin Hanes, Ph.D.

Scientific Advisory Board Member
Dr. Hanes is the Lewis J. Ort professor of ophthalmology at the Wilmer Eye Institute at Johns Hopkins University, with secondary appointments in chemical & biomolecular engineering, environmental health sciences, neurosurgery and oncology. A globally recognized leader of research at the interface of nanotechnology and medicine, Dr. Hanes focuses his research on the delivery of genetic medicines, characterization of mucus and design, and testing of nanoparticles that can penetrate mucus barriers. His lab recently discovered methods to make drug- and gene-loaded particles that efficiently penetrate mucus barriers, which may allow for more effective therapies for eye diseases.

Trisha Millican
ReCode Therapeutics

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.

Dean J. Mitchell

Chairman, Board of Directors

Mr. Mitchell currently serves as chairman of the Board of Directors of Praxis Precision Medicines, Inc., an appointment he has held since 2020, and as a board director for Theravance Biopharma, Inc. (since 2014) and Precigen (since 2009). Mr. Mitchell previously served as board chairman of Kinnate Biopharma, Inc. until its sale in 2024 and as board director for ImmunoGen, Inc. until its sale in 2024. He served as executive board chairman for Covis Pharma Holdings until its sale in 2020 and board chairman of PaxVax Corporation until its sale in 2018.

Mr. Mitchell previously held the position of president and chief executive officer at Lux Biosciences, Inc., a biotechnology company focusing on the treatment of ophthalmic diseases, at Alpharma, Inc., a publicly traded specialty pharmaceutical company until its acquisition by King Pharmaceuticals, Inc., and at Guilford Pharmaceuticals, Inc., a publicly traded pharmaceutical company focused on oncology and acute care, until its acquisition by MGI Pharma Inc. He previously held executive roles at Bristol Myers Squibb and GlaxoSmithKline. Mr. Mitchell earned a B.A. from City University London and a B.Sc. in biology from Coventry University.

Rana Al-Hallaq, Ph.D.

Pfizer Ventures

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.