Moderna and the Institute for Life Changing Medicines Announce a New Collaboration to Develop an mRNA Therapeutic for Ultra-Rare Disease, Crigler-Najjar Syndrome Type 1
September 07 2021 - 8:30AM
Business Wire
Moderna to provide investigational mRNA CN-1
therapy free of charge
Moderna, Inc., (Nasdaq: MRNA) a biotechnology company pioneering
messenger RNA (mRNA) therapeutics and vaccines and the nonprofit
Institute for Life Changing Medicines (ILCM), today announced a new
collaboration to develop a new mRNA therapeutic (mRNA-3351) for
Crigler-Najjar Syndrome Type 1 (CN-1), an ultra-rare disease. It is
estimated that there are only approximately 70-100 known cases of
CN-1 in the world. The goal of the collaboration is to make an mRNA
therapy for the treatment of CN-1 available at no cost to
patients.
Under the terms of the agreement, Moderna will license mRNA-3351
to ILCM with no upfront fees, and without any downstream payments.
ILCM will be responsible for the clinical development of mRNA-3351.
ILCM plans to initiate clinical studies of mRNA-3351 in 2022.
Crigler-Najjar Syndrome Type 1 (CN-1) is an ultra-rare
genetically inherited disorder caused by the mutation in the UGT1A1
gene in which bilirubin, a substance made by the liver cannot be
broken down. The syndrome occurs when the protein that normally
converts bilirubin into a form that can be easily removed from the
body does not work properly. Without this enzyme, bilirubin can
build up in the body and lead to jaundice and damage to the brain,
muscles and nerves. The symptoms become apparent shortly after
birth and can be life-threatening.
Current standard of care treatments rely on phototherapy
treatments of up to 12 hours a day throughout life. The only
definitive treatment is liver transplant that is associated with
its own set of side effects and risk of death.
“At Moderna, we believe that mRNA therapies have the potential
to profoundly impact rare disease patients and their families.
Ultra-rare diseases are always a challenge for our industry given
the very small number of patients who could benefit from the
medicine,” said Stéphane Bancel, Chief Executive Officer of
Moderna. “We decided that rather than charge a high price for the
medicine candidate, which is not aligned with our values, we would
rather give it away for free. In this innovative partnership, the
Institute for Life Changing Medicines will not pay Moderna an
upfront fee or any downstream payments. Moderna will also provide
the mRNA-3351 material free of charge. mRNA-3351 is a therapeutic
candidate for Crigler-Najjar Syndrome Type 1 and our goal is to
bring new hope for patients and their families.”
“Children with this disease can be stigmatized for being
severely jaundiced while living in fear of experiencing a
life-threatening episode of kernicterus,” said James M. Wilson, MD,
PhD, Co-Founder and Chief Scientific Officer of the Institute and
Director of the Orphan Disease Center, Perelman School of Medicine,
University of Pennsylvania. “The mRNA therapeutic platform of
Moderna has the potential to not only treat but to prevent these
lethal metabolic crises. This partnership with Moderna could serve
as a model for developing life changing medicines for those living
with rare diseases when traditional business models for drug
development fall short.”
“We are energized to collaborate with Moderna to advance an mRNA
for treating Crigler-Najjar,” said Alex Karnal, Co-Founder and
Chief Executive Officer of The Institute for Life Changing
Medicines. “At the Institute, we believe that a healthy life is a
basic human right, and we are fighting to improve the lives of all
people suffering from less common but still devastating diseases.
We are grateful for Moderna’s support of our novel nonprofit
business model by providing our first medicine aimed at
transforming the lives of people suffering from Crigler-Najjar
syndrome around the world.”
About mRNA-3351
mRNA-3351 encodes for the human UGT1A1 and is designed to
restore the missing or dysfunctional proteins that causes
Crigler-Najjar Syndrome Type 1. The proposed therapeutic uses the
same proprietary LNP formulation as Moderna’s antibody against
chikungunya virus (mRNA-1944), propionic acidemia (mRNA-3927), and
methylmalonic acidemia (mRNA-3704) programs. mRNA-3351 has been
granted Rare Pediatric Disease designation by the U.S. FDA.
About The Institute for Life Changing Medicines
The Institute for Life Changing Medicines believes that a
healthy life is a basic human right. The Institute identifies,
develops, and promotes access to life changing medicines that are
designed to treat small populations of patients and are therefore
not considered commercially viable. The organization’s pipeline of
next generation medicines includes cell and gene therapies for the
treatment of rare diseases. Visit
https://www.lifechangingmedicines.org/ to learn more.
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, 24 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 www.modernatx.com.
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 regarding: the Company’s collaboration
with the Institute for Life Changing Medicines on the development
of an mRNA therapeutic (mRNA-3351) for Crigler-Najjar Syndrome Type
1; the structure of the collaboration; and the Company’s approach
to ultra-rare diseases. 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 www.sec.gov. 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.
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The Institute for Life Changing Medicines
Media: Contact: Anshul Mangal Position: Chief Strategy Officer
Email: anshulmangal@projectfarma.com
Moderna
Media: Colleen Hussey Director, Corporate Communications
617-335-1374 Colleen.Hussey@modernatx.com
Investors: Lavina Talukdar Senior Vice President & Head of
Investor Relations 617-209-5834 Lavina.Talukdar@modernatx.com
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