MedPath

Cystic Fibrosis Foundation Invests $15 Million in Recode Therapeutics for CF Gene Editing

• The Cystic Fibrosis Foundation is investing $15 million in Recode Therapeutics to advance gene-editing medicines for cystic fibrosis (CF) patients without effective treatment options. • Recode Therapeutics collaborates with Intellia Therapeutics, developing a strategy to alter the genetic code, creating a healthy CFTR gene version for functional protein production. • The funding supports the development of up to three gene-correction programs, initially focusing on CF mutations not addressed by existing therapies. • Recode's RCT2100, an inhaled mRNA therapy delivering a healthy CFTR version to lung cells, is currently in Phase 1 clinical trials, expanding treatment options for all CF patients.

The Cystic Fibrosis Foundation is investing up to $15 million in Recode Therapeutics to accelerate the development of novel gene-editing medicines for individuals with cystic fibrosis (CF) who lack effective treatment options. This investment aims to address the unmet needs of CF patients with specific mutations.
Shehnaaz Suliman, MD, CEO of Recode, expressed gratitude for the CF Foundation's support, emphasizing the potential of their gene correction approach to significantly advance research in collaboration with Intellia and develop a transformative CF treatment.

Targeting the Root Cause of CF

Cystic fibrosis is caused by mutations in the CFTR gene, which provides instructions for making the CFTR protein. The absence of functional CFTR leads to the production of thick, sticky mucus that accumulates in organs, causing most of the disease's symptoms. While CFTR modulators have revolutionized care for many CF patients by improving the functionality of the mutated CFTR protein, they are only effective for individuals with specific mutations.

Gene Editing Approach

Recode, in collaboration with Intellia Therapeutics, is pioneering a new treatment strategy that alters the genetic code in patients' cells to create a healthy version of the CFTR gene, enabling functional protein production. This approach bypasses the limitations of CFTR modulators, potentially benefiting patients ineligible for modulator therapy. The project combines Intellia's gene-editing technology with Recode's delivery technology to introduce gene-editing machinery into patient cells. The funding will support the development of up to three gene-correction programs, initially targeting CF mutations not adequately treated by existing therapies.

Expanding Therapeutic Options

This investment marks the second major contribution from the CF Foundation to Recode. Previously, the foundation invested up to $15 million to support the development of RCT2100, an experimental mRNA therapy currently in clinical testing. Unlike the gene-editing approach, RCT2100 is an inhaled therapy designed to deliver a healthy version of CFTR messenger RNA (mRNA) to lung cells. By delivering healthy CFTR mRNA, RCT2100 aims to enable the production of functional CFTR in lung cells, irrespective of the patient's underlying mutation.
RCT2100 is currently being evaluated in a two-part Phase 1 clinical trial (NCT06237335). The first part involves testing the therapy in healthy volunteers, while the second part is testing it in people with CF. Patient dosing has commenced, with ongoing recruitment in New Zealand and the Netherlands. Additional sites in the U.S., the U.K., and France are expected to enroll patients in the future.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

Recode Therapeutics' RCT-2100 Shows Promise for Cystic Fibrosis Treatment

Recode Therapeutics is developing RCT-2100, an mRNA-based therapy, for cystic fibrosis, utilizing their SORT lipid nanoparticle platform.
RCT-2100 delivers mRNA encoding the CFTR protein via inhalation, addressing the underlying genetic defect in cystic fibrosis patients.

Spirovant Sciences Doses First Patient in Phase 1/2 Trial of SP-101 Gene Therapy for Cystic Fibrosis

Spirovant Sciences has initiated dosing in a Phase 1/2 clinical trial (SAAVe) evaluating SP-101, an inhaled gene therapy for cystic fibrosis (CF), in adults aged 18-65.
The SAAVe trial (NCT06526923) aims to enroll 15 CF patients with stable disease and a ppFEV1 between 50% and 100% to assess the safety and efficacy of SP-101.

Intellia's NTLA-2001 Shows Promise in Early ATTR-CM Trial

Intellia Therapeutics' NTLA-2001 demonstrates potential in stabilizing or improving outcomes for transthyretin amyloidosis with cardiomyopathy (ATTR-CM) patients.
Phase I study data reveals a median five-meter improvement in the six-minute walk test after one year of NTLA-2001 treatment.

Spirovant Sciences Doses First Patient in Cystic Fibrosis Gene Therapy Trial

Spirovant Sciences has commenced its SAAVe Phase I/II study, dosing the first patient with SP-101, a gene therapy for cystic fibrosis, in combination with an augmenter.
SP-101, an inhalable adeno-associated virus (AAV) gene therapy, is engineered for tropism in human airway epithelia to enhance CFTR transgene expression.

FDA Approves PTC Therapeutics' Kebilidi, First Direct-to-Brain Gene Therapy

The FDA has approved PTC Therapeutics' Kebilidi (eladocagene exuparvovec-tneq) for aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder.
Kebilidi is the first gene therapy approved by the FDA that is administered directly into the brain, specifically targeting the putamen region.

FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Six Gene Therapies

The FDA has granted Rare Pediatric Disease (RPD) and Orphan Drug Designations (ODD) to six investigational gene therapies developed under the Accelerating Medicines Partnership (AMP) Bespoke Gene Therapy Consortium (BGTC).
These designations aim to incentivize the development of gene therapies for rare diseases affecting children, offering financial benefits such as Priority Review Vouchers and tax credits.

SORT Nanoparticles Enable Durable In Vivo Gene Editing in Lung Stem Cells

Researchers developed selective organ targeting (SORT) nanoparticles for targeted delivery of gene-editing molecules to the lungs.
A one-time treatment with SORT nanoparticles achieved durable gene editing in mouse lungs for nearly two years.

Bicara, Zenas, and MBX Bio Launch IPOs, Raising $703M for Clinical Trials

Bicara Therapeutics raised $315 million to fund a pivotal trial of ficerafusp alfa for head and neck squamous cell carcinoma (HNSCC).
Zenas Biopharma secured $225 million to advance obexelimab, a bifunctional antibody, through Phase 3 trials for IgG4-related disease and Phase 2 trials for multiple sclerosis, lupus, and wAIHA.

RDCA-DAP Boosts Cystic Fibrosis Research with New Data Sets

The RDCA-DAP has acquired new datasets from the Cystic Fibrosis Therapeutics Development Network to accelerate cystic fibrosis (CF) research and therapy development.
The data sharing initiative seeks to enhance understanding of CF progression, design new clinical outcome measures and biomarkers, and develop disease models.

Passage Bio Receives FDA Positive Feedback for Expanding Gene Therapy Trial to Include FTD-C9orf72 Patients

Passage Bio has received positive feedback from the FDA to expand its phase 1/2 upliFT-D clinical trial to include patients with frontotemporal dementia (FTD) due to C9orf72 mutations, following encouraging data from the trial's first cohort. The gene therapy PBFT02 aims to increase progranulin levels in the central nervous system, showing promising results in initial clinical data.

Reference News

[1]
CF Foundation invests $15M in Recode Therapeutics to boost CF gene editing
cysticfibrosisnewstoday.com · Nov 20, 2024

The Cystic Fibrosis Foundation is investing up to $15 million in Recode Therapeutics to develop gene-editing medicines f...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy