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A Clinical Study for People with Cystic Fibrosis (CF)

Who Have Specific Mutations in the CFTR Gene, Which Are the Cause of CF

This clinical study is exploring a potential new inhaled messenger RNA (mRNA) treatment called RCT2100 that may improve the function of the CFTR protein in the lungs, getting at the root cause of CF lung disease rather than just controlling symptoms.

Here, you will find more information about the study, investigational medicine, and how you, or someone you know, can participate.

About Cystic Fibrosis

CF is a progressive genetic disease that causes persistent lung infections and respiratory failure. It is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and impacts approximately 130,000 people worldwide.

The absence or dysfunction of the CFTR protein results in a defect in airway hydration, which leads to excessive mucus buildup in the lungs. It also creates a mucociliary clearance defect, recurrent infections, inflammation, respiratory failure, and other complications.

Despite the availability of CFTR modulators that work for 90% of people with CF, many will not be able to take the medication due to side effects, lack of response, or other reasons. About 10% of the CF community have genetic mutations that do not benefit from these therapeutics.

CF impacts approximately 130,000 people worldwide. 90% have responsive genotypes to CFTR modulators, and 10% (13,000 people) do not.

Why this Study is Important

No CFTR modulator therapies are currently approved to treat individuals with certain mutations in the CFTR gene because these mutations prevent the production of any CFTR protein.

Without CFTR protein, there is nothing available for the therapy to correct. This study aims to find out whether the investigational study medicine is safe and may work as a potential new treatment option for people with CF who are not eligible for, or are eligible but not currently taking, CFTR modulator therapy.

To learn more, contact:

[email protected]

How the Clinical Study Medicine Works

ReCode is developing RCT2100, an investigational inhaled therapy for cystic fibrosis (CF) based on messenger RNA (mRNA). This treatment is designed to deliver CFTR mRNA directly to target cells in the lungs, instructing them to produce a functional version of the CFTR protein.

Unlike current CFTR modulators that work on existing defective proteins by either correcting or potentiating that protein, RCT2100 is designed to allow the cells to make new functional CFTR protein.

By providing CFTR mRNA to individuals with CF who are missing this protein, the therapy aims to address the underlying cause of the disease, potentially improving CFTR protein function in the lungs rather than just managing symptoms.

How the Clinical Study Medicine is Given

The investigational medicine, RCT2100, will be given using a nebulizer — a medical device that converts liquid medication into a mist for inhalation.

1 Screening

There will be a 2–4-hour office visit to determine your eligibility to participate and an opportunity for you to ask any questions to see if this study is right for you.

2 Treatment

If you are eligible, you will receive inhaled doses of RCT2100.

3 Follow-up

After your last dose, there will be a 24-week follow-up period, which includes a final outpatient visit.

Study tests and procedures

Throughout the study, you will have certain exams and tests, including:

Physical Exam

Vital Signs Measurement

Electrocardiogram
A non-invasive test to record the heart’s electrical activity

Chest X-Ray
(if you have not had one within six months)

Pulse oximetry
A non-invasive test that measures oxygen in the blood

Spirometry
A non-invasive test to measure how much and how fast you can move air into and out of your lungs and how well your lungs are exchanging gases

Blood and Urine Tests

Full details will be provided for your consideration prior to completing and signing an informed consent which confirms you fully understand the process and implications of taking part in the study.

Eligibility

Who Can Participate
  • Those who are 18-65 years old
  • Those with a diagnosis of CF
  • People with CF not eligible for treatment with CFTR modulators based on having mutations of the CFTR gene or people with CF who are eligible, but not able to tolerate CFTR modulators
  • Anyone with a forced expiratory volume (FEV1) greater than 40% predicted
  • Those willing to use contraception
Who Cannot Participate
  • Those who are pregnant or breastfeeding
  • Anyone who has received treatment with a CFTR modulator in the last 12 weeks
  • Those who have had a CF pulmonary exacerbation in the last 4 weeks
  • Anyone with a lung infection caused by organisms including B. cenocepacia, B. dolosa or M. abscessus
  • An oxygen saturation <94% on room air

FAQs

What is a clinical research study?

A clinical research study is a carefully designed investigation to assess the safety, effectiveness, and potential benefits of medical treatments, interventions, or procedures in humans. These studies are essential for advancing medical knowledge and improving patient care.

Participants in a clinical study may receive new investigational treatments and are closely monitored to observe their responses and outcomes. The studies follow strict ethical guidelines and regulatory requirements to ensure the safety and rights of participants.

By participating in a clinical research study, people with CF contribute to developing new medical therapies and may gain access to innovative treatments that might not yet be widely available.

Many study sites are enrolling in the U.S. and U.K., as well as in France and the Netherlands. Click here to find a list of all available study sites.

If you are eligible and decide to participate in the study, the research team will share more information about the time expected from you during the study.

There are three parts to the study:

  1. Screening period: involves a 2–4-hour office visit to determine your eligibility to participate and an opportunity for you to ask any questions to see if this study
    is right for you.
  2. Treatment period: will take 4 weeks or 12 weeks where you will receive RCT2100
  3. Follow-up period: will take 24 weeks.

If you participate, you can expect to be in the study for up to 40 weeks (~10 months).

Participants will be reimbursed for travel expenses and will receive additional per-visit stipends (US).
There will be no cost for you to participate. ReCode will pay for all required tests, procedures, travel, and accommodations

Participating may involve some potential risks, which may occur from the investigational study medicine or the tests.

You will receive an informed consent form describing the full risks involved before agreeing to participate in the study.

The research team will also discuss risks with you and answer any questions that you may have about the study.

Participating in a clinical research study for CF may contribute to the advancement of medical science, helping to develop new treatments for CF that can benefit others in the future.

With the research team’s approval, this trial offers a home visit option. This option allows for dosing at your home, carried out by a research nurse trained to administer RCT2100 after you receive your first two doses. The research nurse will arrive one hour before dosing and remain at your home one-hour post-dose.

The withdrawal process will be discussed in detail at the initial consultation.

The research team at the trial site will oversee your care during the study. If the CF doctor leading the study is not a member of your usual CF Specialist Team, they will closely liaise with them to oversee your care during your participation period. They will work with the study team to ensure the clinical trial does not conflict with any medications or treatments you may currently be receiving.
Participants in the RCT2100-101 trial should not be excluded from other trials after an appropriate wash-out period (as defined in the other study protocols for the trials participants may be interested in).
Please refer to the ReCode privacy policy here.

To find out more, contact:

[email protected]
Dividing Line
Now Enrolling
ReCode CF mRNA RCT2100-101 Study
If you’re interested in participating or would like more information, please contact us at [email protected]
CONTACT US

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.

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.