Moderna Announces First Patient Dosed in Phase 1/2 Study of mRNA-3705 for Methylmalonic Acidemia

August 16, 2021

Moderna Announces First Patient Dosed in Phase 1/2 Study of mRNA-3705 for Methylmalonic Acidemia

August 16, 2021 at 8:57 AM EDT

mRNA-3705 is Moderna’s second rare disease program to enter clinical studies

mRNA-3705 granted Orphan Drug and Rare Pediatric Disease designation by the U.S. FDA

MMA associated with significant mortality and morbidity; there are no approved therapies

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aug. 16, 2021-- Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced the first patient has been dosed in the Phase 1/2 study evaluating the safety and tolerability of mRNA-3705, its investigational mRNA therapeutic for methylmalonic acidemia (MMA), administered via intravenous infusion in patients with isolated MMA due to MUT deficiency.

“We would like to thank Dr. Santra and the whole team in Birmingham for their efforts and collaboration to achieve this milestone moment of dosing the first MMA patient with our mRNA therapeutic,” said Ruchira Glaser, M.D., Sr. Vice President and Therapeutic Area Head, Rare Disease, Autoimmune & Cardiovascular. “This is another step forward in Moderna’s mission to deliver on the promise of mRNA science to create a new generation of transformative medicines for patients.”

“We are delighted to have been able to treat the first patient in the world with this new medicine here in Birmingham,” Saikat Santra, M.D., Pediatric Inherited Metabolic Medicine Consultant, Clinical Inherited Metabolic Disorders at Birmingham Women’s and Children’s NHS Foundation Trust. “We sincerely hope that it brings this brave patient, and many more like them, a brighter future free of the restrictions of this terrible disease.”

Methylmalonic acidemia is a rare, life-threatening, inherited metabolic disorder that is most commonly (approximately 60% of cases) caused by a deficiency in the mitochondrial enzyme methylmalonic-CoA mutase (MUT). This deficiency can lead to metabolic crises due to a toxic buildup of acids in the body and progresses into multi-organ disease. As a result, MMA is associated with significant mortality and morbidity, and there are no approved therapies. Standard of care includes dietary and palliative measures. Currently, liver or combined liver and kidney transplant is the only effective treatment.

Moderna recognizes the impact of rare diseases on patients and their families, particularly when the disease lacks effective treatment options. In addition to MMA, Moderna is working to advance mRNA therapeutics that restore the activity of missing enzymes responsible for other rare diseases, such as propionic acidemia (PA), glycogen storage disease type 1a (GSD1a) and phenylketonuria (PKU). Moderna has active clinic programs in five different therapeutic areas: infectious disease, oncology, cardiovascular, rare disease and autoimmune disease.

About the Phase 1/2 Study

The Phase 1/2 study, called the “Landmark study,” is an adaptive, open-label study is designed to evaluate the safety and tolerability of up to five different dosing regimens of mRNA-3705 administered via intravenous infusion in patients one year and older with isolated methylmalonic acidemia due to methylmalonyl-CoA mutase (hMUT). Upon establishment of an optimized dose based on safety and pharmacological data, additional patients maybe enrolled in an optional expansion cohort.

About mRNA-3705

mRNA-3705 is designed to instruct the body to restore the missing or dysfunctional proteins that cause MMA and consists of mRNA encoding human MUT, the mitochondrial enzyme commonly deficient in MMA, encapsulated within Moderna’s proprietary lipid nanoparticle (LNP). mRNA-3705 uses the same proprietary LNP formulation as the Company’s antibody against chikungunya virus (mRNA-1944) and propionic acidemia (mRNA-3927) programs. mRNA-3705 has been granted Orphan Drug and Rare Pediatric Disease designation by the U.S. Food and Drug Administration (FDA). Moderna owns worldwide commercial rights to mRNA-3705.

About Moderna

In 10 years since its inception, Moderna has transformed from a science research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across six modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for both clinical and commercial production at scale and at unprecedented speed. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna’s capabilities have come together to allow the authorized use of one of the earliest and most-effective vaccines against the COVID-19 pandemic.

Moderna’s mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Today, 23 development programs are underway across these therapeutic areas, with 15 programs having entered the clinic. Moderna has been named a top biopharmaceutical employer by Science for the past six years. To learn more, visit

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: the Company’s development of an investigational therapeutic for methylmalonic acidemia (MMA) (mRNA-3705); the conduct of clinical trials for mRNA-3705; and the Company’s efforts to develop therapies for other rare diseases, including propionic acidemia, glycogen storage disease type 1a and phenylketonuria. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading “Risk Factors” in Moderna’s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SEC’s website at Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna’s current expectations and speak only as of the date hereof.

Colleen Hussey
Director, Corporate Communications

Lavina Talukdar
Senior Vice President & Head of Investor Relations

Source: Moderna, Inc.