Pharma Focus Europe

Nanite and SalioGen Therapeutics Collaborate on Non-Viral Nanoparticle for Cystic Fibrosis Gene Therapy Targeting Lungs

Thursday, May 30, 2024

Nanite, Inc. and SalioGen Therapeutics have recently entered into a collaborative research venture aimed at utilizing Nanite's SAYER™ delivery platform. The goal is to develop a lung-targeting polymer nanoparticle (PNP) using SalioGen's Gene Coding™ technology. This PNP is intended to integrate the cystic fibrosis transmembrane conductance regulator (CFTR) gene directly into lung cells. CFTR gene mutations lead to cystic fibrosis (CF), a progressively debilitating condition affecting the lungs, digestive system, and other vital organs.

Nanite's SAYER platform combines AI-driven polymer design with in vivo screening to develop PNPs tailored for tissue-specific delivery. SAYER has shown promising results in targeting the lungs, potentially overcoming limitations associated with viral vectors and lipid nanoparticles.

SalioGen's Gene Coding technology utilizes a bioengineered transposase enzyme to deliver an optimized construct containing the entire gene of interest to target cells' genome, regardless of its size. In vitro experiments have successfully demonstrated the integration of the full-length CFTR gene into human lung cells.

As per the collaboration agreement, Nanite will design a bioabsorbable polymer delivery vehicle for SalioGen's Gene Coding technology. Sean Kevlahan, Nanite's co-founder, and CEO, emphasizes the significance of this partnership in developing safe polymeric nanoparticles for CF treatment. He also highlights Nanite's ongoing CF research supported by the Cystic Fibrosis Foundation's Path to a Cure.

SalioGen Therapeutics, expresses enthusiasm for the collaboration, stating its potential to accelerate the impact of genetic medicine for CF patients. He hopes to develop a non-viral genetic medicine directly delivered to the lungs, providing a permanent therapy for individuals with CF, regardless of their specific mutation.



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