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miRecule today announced a strategic collaboration and exclusive licensing agreement with Sanofi to develop and commercialize antibody RNA conjugates (ARCs) for the treatment of facial scapulohumeral muscular dystrophy (FSHD).
This is the first authorized transaction on miRecule's proprietary DREAmiR platform, with a total value of nearly $400 million
.
FSHD is an inherited, progressive atrophic disorder that commonly affects the muscles of the face, shoulder blades and upper arms, and is the second most common muscular wasting disorder that remains untreated
.
People with FSHD usually develop weakness and atrophy
of the eye, mouth, shoulder, upper arm, and calf muscles before the age of 20.
As the disease worsens, FSHD may spread to the abdominal and hip muscles, and about 20% of patients will need to use
a wheelchair before the age of 50.
The FSH Society estimates that about 870,000 people worldwide have the disease
.
FSHD usually develops slowly and is not fatal, but it can seriously affect the quality of life of patients, and some of them even require respirators to live
their lives normally.
About 95% of FSHD patients are caused
by abnormal expression of the DUX4 gene due to a variant of chromosome 4.
by abnormal expression of the DUX4 gene due to a variant of chromosome 4.
miRecule's proprietary, genome-based DREAmiR discovery platform identifies key RNA targets for drug development
in specific populations.
miRecule has developed proprietary RNA therapies with improved pharmacological properties that can be conjugated to antibodies for delivery to target tissues
.
For example, miRecule's pilot MC-30 replaces microRNA-30 (miRNA-30), which has tumor suppressive activity in head and neck cancer, and about half of head and neck cancer patients lose miRNA-30 expression
.
miRecule's second project is MC-DX4, an RNA therapy that eliminates the expression
of the DUX4 gene in FSHD patients.
The collaboration will combine miRecule's RNA therapy targeting DUX4 with Sanofi's proprietary muscle-targeting NANOBODY technology
.
The two molecules will be combined with chemical formulations through miRecule's NAVIgGator conjugation technology to become ARC drugs
.
This potential "best-in-class" drug may have the effect of altering the disease process, and is expected to restore the debilitating, abnormal aging process
caused by disease progression in FSHD patients.
.
The two molecules will be combined with chemical formulations through miRecule's NAVIgGator conjugation technology to become ARC drugs
.
Under this agreement, miRecule will grant Sanofi exclusive rights
to FSHD therapies worldwide.
The two companies will collaborate on research in the pilot project screening
.
After the pilot project selection is completed, Sanofi will be responsible for the preparation of new drug clinical trial (IND) applications, as well as subsequent global development and commercialization activities
.
miRecule will receive an upfront payment from Sanofi and be eligible for future milestone payments, estimated to reach nearly $400 million
.
"We are excited
to be able to enter the first large licensing transaction.
Our goal since the beginning of this project has been to develop a therapy
that enables people with FSHD to lead normal lives.
The DREAmiR discovery platform focuses on patient-centered therapy development and is informed by a solid scientific foundation
.
We believe Sanofi shares our values of creating life-changing therapies that will bring this DUX4-targeted RNA therapy to patients
.
" ”
