ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT1100 for the Treatment of Primary Ciliary Dyskinesia
27 June 2024 - 9:30PM
Business Wire
ReCode Therapeutics, a clinical-stage genetic medicines company
using tissue-specific delivery to power the next wave of mRNA and
gene correction therapeutics, today announced that the U.S. Food
and Drug Administration (FDA) has granted Orphan Drug Designation
for RCT1100 for the treatment of primary ciliary dyskinesia (PCD).
RCT1100 is a novel investigational therapy being developed to treat
PCD caused by pathogenic mutations in the DNAI1 gene.
PCD is a rare and progressive genetic disease characterized by
the dysfunction of cilia, which are microscopic hairlike structures
that line the respiratory tract. Defective ciliary function results
in the inability to clear mucus from the airways. As a result,
bacteria and viruses become trapped in the airways, leading to
chronic infections and compromised respiratory function, with a
small percentage of patients requiring a lung transplant. Mutations
in the DNAI1 gene account for approximately seven percent of PCD
cases.
“PCD is a rare genetic lung disorder that currently has no
FDA-approved treatments; therefore, receiving this designation is
an important recognition for the PCD patient community," said
Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of
ReCode Therapeutics. "We are actively enrolling PCD patients in our
single-dose study across the U.S. and UK and look forward to
sharing an update when enrollment is completed.”
ReCode’s proprietary Selective Organ Targeting (SORT) lipid
nanoparticle (LNP) delivery platform was used to develop RCT1100,
an inhaled mRNA therapy designed to deliver DNAI1 mRNA to target
airway cells. The DNAI1 mRNA is translated into the DNAI1 protein
inside cells, which is intended to establish proper ciliary
function by addressing the underlying cause of the disease. In
preclinical studies, the company’s DNAI1 mRNA led to efficient
production of the DNAI protein and rescue of ciliary function in
relevant models of PCD.
ReCode is currently enrolling patients in Part B of a Phase 1,
open-label study designed to assess the safety and tolerability of
a single ascending dose of inhaled RCT1100 administered with a
nebulizer in patients with PCD caused by pathogenic mutations in
the DNAI1 gene. For more information, please visit
clinicaltrials.gov.
About Primary Ciliary Dyskinesia
Primary ciliary dyskinesia (PCD) is a rare genetic disease
characterized by deficient mucociliary clearance (MCC), chronic
respiratory tract infections, bronchiectasis, and declining
respiratory function. Mutations in more than 50 genes result in
dysfunctional cilia and loss of MCC. PCD is a progressive disorder,
meaning it will get worse over time, for which no cure or
disease-modifying treatments currently exist. PCD is estimated to
affect at least 1 in 7,500 people across diverse ethnicities, with
approximately 45,000 patients in the U.S. Due to the lack of
widespread screening, approximately 80% of people with PCD are
thought to be undiagnosed.
ReCode has launched ThinkPCD.com, a program that provides
genetic testing to PCD patients who qualify at no cost. Patients
can enroll directly or through a healthcare professional who can go
to the website to determine if their patients are eligible for
genetic testing through the program.
About ReCode Therapeutics
ReCode Therapeutics is a clinical-stage genetic medicines
company using precision delivery to power the next wave of mRNA and
gene correction therapeutics. ReCode’s proprietary Selective Organ
Targeting (SORT) lipid nanoparticle (LNP) platform enables highly
precise and targeted delivery of genetic medicines directly to the
organs, tissues, and cells implicated in disease, enabling improved
efficacy and potency. ReCode’s lead programs include RCT1100 for
the treatment of primary ciliary dyskinesia caused by pathogenic
mutations in the DNAI1 gene, and RCT2100 for the treatment of the
10-13% of cystic fibrosis patients who have Class I or other
mutations in the CFTR gene that do not respond to currently
approved CFTR modulators. RCT1100 and RCT2100 are inhaled
disease-modifying mRNA-based therapies formulated using the SORT
LNP delivery platform. ReCode is expanding its pipeline to develop
potential therapies for other rare and common genetic diseases,
including musculoskeletal, central nervous system, liver, and
infectious disease indications.
ReCode has been recognized by the San Francisco Business Times
and Silicon Valley Business Journal as a Best Place to Work. For
more information, visit www.recodetx.com and follow us on LinkedIn
and Instagram.
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version on businesswire.com: https://www.businesswire.com/news/home/20240627289896/en/
Investors: Anne Marie Fields
Stern IR annemarie.fields@sternir.com IR@recodetx.com
Media: Erica Jefferson SVP,
Corporate Affairs ReCode Therapeutics ejefferson@recodetx.com
650-629-7965
Tara Cooper The Grace Group tara@gracegroup.us 650-303-7306