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ReCode Therapeutics Presents Preclinical Data from Inhaled mRNA Therapeutic Program in Primary Ciliary Dyskinesia (PCD) in Three Posters at the ATS 2022 International Conference

ReCode’s Selective Organ Targeting (SORT) lipid nanoparticle (LNP) formulation of DNAI1 mRNA was successfully and directly delivered to lungs as an inhaled aerosol with no significant exposure to other tissues
 SORT LNP-formulated DNAI1 mRNA demonstrates long-lasting functional recovery and is well-tolerated with rapid clearance of LNP lipids allowing repeat administration 
ReCode to present new preclinical data from its mRNA-based cystic fibrosis program on Wednesday, May 18th 

Menlo Park, Calif. and Dallas, Texas – May 15, 2022 – ReCode Therapeutics, a biopharmaceutical company powering the next wave of genetic medicines through superior delivery today presented new preclinical data from the company’s inhaled mRNA-based molecular therapy program for the treatment of primary ciliary dyskinesia (PCD) in three posters at the American Thoracic Society (ATS) 2022 International Conference, taking place May 13-18, 2022 in San Francisco. The company’s mRNA-based program in PCD uses ReCode’s first-in-class Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform, which enables the targeting of organs and tissues beyond the liver via a variety of administration routes.

There are currently no approved treatments for primary ciliary dyskinesia (PCD), a rare genetic disease with inherited mutations that cause the loss of ciliary activity (and therefore the loss of normal mucus clearance) in the airways. Patients with PCD have a high burden of morbidity with chronic respiratory infections, bronchiectasis, and often develop respiratory failure.

ReCode is developing a disease-modifying mRNA-based approach for the treatment of PCD caused by mutations in DNAI1, an essential gene for ciliary movement. Using the company’s proprietary delivery platform, DNAI1 mRNA is formulated in ReCode’s SORT LNPs, nebulized, and delivered as an aerosol directly into the airway. The intent is for the mRNA delivery to lead to DNAI1 protein production in target cells and therefore enable rescue of ciliary function.

“These promising new data demonstrate that our SORT LNP-formulated DNAI1 mRNA can be precisely delivered directly to the intended cells within the lungs, without significant exposure to other tissues. Importantly, treatment was shown to be well-tolerated, allowing for repeat administration. Additional preclinical data show that our DNAI1 mRNA has low immune reactivity, rescues ciliary function, and results in long-lasting functional recovery,” said David Lockhart, Ph.D., Chief Scientific Officer and President, ReCode Therapeutics. “The totality of data presented at ATS today reflects the broad potential of our novel SORT LNP platform to deliver highly targeted mRNA-based therapies and supports the continued development of inhaled mRNA as a promising disease-modifying therapy for primary ciliary dyskinesia, a rare disease with no approved treatments.”

New data presented today at ATS included:

  • Results from a non-human primate model show that ReCode’s SORT LNP-formulated mRNA is well-tolerated and can be nebulized and delivered directly to the lungs as an inhaled aerosol without significant exposure to other tissues. Robust delivery of DNAI1 mRNA to the lung and expression of human DNAI1 protein in relevant target cell types, including ciliated, club, and basal cells, was observed. Study results showed rapid clearance of LNP lipids which suggests the potential for well-tolerated repeat administration.
  • In a proof-of-activity study using the human bronchial epithelial (hBE) cell-based PCD model, SORT LNP-formulated DNAI1 mRNA delivered as an aerosol successfully rescued ciliary function that was shown to persist for weeks after the last treatment.
  • A preclinical design showed that the optimized DNAI1 mRNA reduced immune reactivity and was well-tolerated.

Full details from the data presented at ATS 2022 can be accessed via the Publications section on ReCode’s website.

ReCode will also present new preclinical data from its mRNA-based therapeutic program for cystic fibrosis on Wednesday, May 18th. These data demonstrate the ability of SORT LNPs to deliver optimized, functional CFTR mRNA as an aerosol directly to the intended cell targets. These preclinical data support further investigation and provide a potential therapeutic approach to address a significant patient population that is not eligible for current CFTR modulator therapy.

Poster Title: Functional Rescue of CFTR by Aerosolized Delivery of Optimized CFTR mRNA Using ReCode-LNPs in Primary Human Bronchial Epithelial Cells Derived from Patients with Cystic Fibrosis
Poster Number: 705
Session Title: D109 – Airway of Interest: Epithelial and Smooth Muscle Function in Health and Disease
Date and Time: Wednesday, May 18, 2022, 12:45 – 2:15 p.m. PT
Location: Room 2009/2011 (West Building, Level 2), Moscone Center
Presenter: Daniella Ishimaru, Principal Scientist, ReCode Therapeutics

About Primary Ciliary Dyskinesia 

Primary ciliary dyskinesia (PCD) is a rare genetic disease characterized by deficient mucociliary clearance (MCC), chronic respiratory tract infections, bronchiectasis, and declining respiratory function. Mutations in more than 40 different genes result in dysfunctional cilia and loss of MCC. PCD is a life-limiting disease with a high burden of morbidity and no disease-modifying treatments available to patients. There are estimated to be more than 87,000 patients affected with PCD across North America and Europe, with more worldwide.

About the SORT LNP Platform

ReCode’s novel Selective Organ Targeting (SORT) lipid nanoparticle (LNP) technology is a modular genetic medicines delivery platform with broad potential applications across a range of organs and tissues, target cells, and therapeutic modalities. ReCode’s proprietary SORT LNP platform enables the flexibility to target specific organs and tissues while de-targeting the liver.

Beyond its highly selective targeting capability, ReCode’s SORT LNP platform is further distinguished by its versatility in both mode of administration and the diversity of genetic cargoes that can be delivered, which include mRNA and gene correction components. Together, these qualities form the foundation for opportunities to unlock the full potential of genetic medicines.

About ReCode Therapeutics

ReCode Therapeutics is a biopharmaceutical company powering the next wave of genetic medicines through superior delivery. ReCode’s Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform is a next-generation LNP delivery technology to target organs and tissues beyond the liver. The SORT LNP platform is the foundation for ReCode’s pipeline of disease-modifying mRNA and gene-correction based therapeutics for genetically defined diseases for which there are few or no current treatments.  ReCode’s lead programs are focused on primary ciliary dyskinesia, and cystic fibrosis caused by Class I mutations. ReCode is leveraging its SORT LNP platform and nucleic acid technologies for mRNA-mediated replacement and gene correction in target cells, including stem cells. For more information, visit www.recodetx.com and follow us on Twitter @ReCodeTx and LinkedIn.

Investor Contact:
Sarah McCabe
Stern IR
[email protected]
[email protected]

Media Contact:
Tara Cooper
The Grace Communication Group
[email protected]
650-303-7306
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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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

Oleg Nodelman

EcoR1 Capital

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.

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.

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.

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.