Modalis Therapeutics: FDA Grants Rare Pediatric Disease Designation to MDL-101 for the Treatment of Congenital Muscular Dystrophy Type 1a (LAMA2-CMD)
30 Septiembre 2024 - 1:05AM
Business Wire
Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883,
CEO: Haru Morita), a leading company pioneering treatments for rare
genetic diseases using its proprietary CRISPR-based epigenome
editing technology, CRISPR-GNDM®, today announced that the U.S.
Food and Drug Administration (FDA) has granted Rare Pediatric
Disease (RPD) designation to MDL-101, a novel precision medicine
being developed for the treatment of congenital muscular dystrophy
type 1a (LAMA2-CMD).
The FDA grants RPD designations to treatments for serious and
life-threatening diseases that primarily affect children under 18
years old and affect fewer than 200,000 people in the U.S. If a
product with RPD designation receives marketing approval, the
company may be awarded a Priority Review Voucher (PRV), allowing
for faster FDA review of another product. Modalis has also
submitted an Orphan Drug application for MDL-101, which is
currently under separate review by the FDA.
“We are pleased that the FDA has recognized our development
efforts for the rare disease and granted us RPD designation. We
have received many requests for our efforts from children and
families around the world suffering from this disease for which
there is currently no treatment, and we feel a mission to respond
to the expectations of patients who are eagerly awaiting the start
of clinical trials as soon as possible,” said Haru Morita, CEO of
Modalis. “This designation from the FDA is proof that our efforts
are rational and achieving results, and we hope to further
accelerate the development of this world's first product using the
cutting-edge technology.”
LAMA2-CMD is a severe, early onset congenital muscular dystrophy
caused by the absence of the LAMA2 protein, which is made up of
more than 3,000 amino acids. Because it cannot be loaded onto an
AAV vector, it is thought that the conventional approach to gene
therapy development is difficult. For this reason, there is
currently no treatment, including gene therapy, that addresses the
underlying cause of LAMA2-CMD.
Modalis’ proprietary CRISPR-GNDM® technology allows for precise
modulation of gene expression without introducing double-strand DNA
breaks. MDL-101, Modalis’ lead candidate for LAMA2-CMD, represents
a first-in-class therapeutic that aims to address the unmet need by
inducing expression of the sister gene LAMA1 in muscle tissues,
thereby compensating for the deficient function of LAMA2.
Guided by its mission, "Every Life Deserves Attention," Modalis
is dedicated to bringing life-changing treatments to patients
suffering from diseases for which no cure currently exists.
About MDL-101
MDL-101 is an experimental, epigenetic editing therapy that is
being developed for the treatment of LAMA2-Congenital Muscular
Dystrophy (LAMA2-CMD). MDL-101 is comprised of a guide nucleotide
targeting LAMA1 gene, a highly homologous sister gene of the
disease-causing gene LAMA2, enzyme-null Cas9 (dCas9) fused with
trans-activating domain driven by a muscle-specific promoter and
coded in a muscle-specific AAV vector. MDL-101 upregulates LAMA1
gene products in patients’ muscle tissue to compensate for
loss-of-function caused by mutation of LAMA2, and therefore has the
potential to provide a one-time, durable treatment to benefit
people living with LAMA2-CMD.
About Modalis:
Modalis Therapeutics develops precision genetic medicines using
epigenome editing technology. Modalis is pursuing therapies for
orphan genetic diseases using its proprietary CRISPR-GNDM®
technology which enables the gene/locus-specific modulation of gene
expression or epigenome editing without the need for DNA cleavage
or altering DNA sequence. Headquartered in Tokyo with all research
and development operations in Waltham Massachusetts, the company is
listed on the Tokyo Stock Exchange’s Growth market. For additional
information, visit www.modalistx.com/en/.
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For further information, please contact: Modalis Therapeutics
Corporation Corporate Communications Sawako Nakamura
media@modalistx.com