Science

Drug Delivery Solutions

Core Technology

Nitto BioPharma offers industry leading solutions for nucleic acid drug delivery. Our novel biodegradable LNPs enable passive or active targeting to specific organs or cells and efficiently deliver the nucleic acid payload into the target cells for activity (silencing or expression of proteins).

Designed to perform competitively compared to the leading benchmarks

Ensure a high level of activity, while exhibiting the best performance for tolerability and safety

Clinically-tested formulations for liver and lung delivery for licensing

Opportunities to license our lipids and LNPs for applications in their therapeutic pipeline development activities

Our research has resulted in lipid-based delivery systems for RNA-based and DNA -based therapies

siRNA or miRNA

mRNA, replicon mRNA, or circular mRNA

DNA

Gene-Editing Cargo (Cas9 + gRNA)

LNP Delivery Systems

Key Features & Capabilities

Nitto develops LNPs to deliver nucleic acid cargo to a variety organ systems including liver, lung, kidney and more.

Our LNPs are a platform technology that utilizes different lipids and components to develop passive or active targeted LNPs

Biocompatible, biodegradable, non immunogenic, non hemolytic, and well tolerated

Scalable, proprietary manufacturing process

LNP Lyophilization Technology for drug product stability – No filtration or premedication required for delivery

IP is licensed as a bundle to enable turn key LNP solutions for delivery for partners

Nitto BioPharma leads in nucleic acid drug delivery Science with biodegradable LNPs, targeting specific cells to deliver and activate nucleic acids.

Nitto's LNP Delivery Systems

Properties & Benefits

Nitto BioPharma offers industry leading solutions for nucleic acid drug delivery. Our novel biodegradable LNPs enable passive or active targeting to specific organs or cells and efficiently deliver the nucleic acid payload into the target cells for activity (silencing or expression of proteins).

Benefits

Greater than 15 unique lipid families ensuring diversity and versatility

Active targeting technology to deliver the LNPs to specific cells via a receptor targeting ligands

Process to manufacture small, uniform nanoparticles with robust process reproducibility and stability

Properties

Millions of potential lipid structures, with over 1000 lipids in inventory

Ionizable and constitutive charged lipids

Specialty lipids and components to enhance activity

Includes lipids that are clinically derisked and have associated developmental support and data

Designed for safety and tolerability

LNP Formulation	Attributes

Mean Particle Size	50 to 100nm (Depends on modality)
Polydispersity Index	≤ 0.2
Encapsulation Efficiency	≥ 90
Zeta Potential in PBS	Neutral
Dosage Form	Lyophile
Manufacturing Process	Proprietary process
Storage Conditions	Refrigerated 2-8 °C to -20 °C
Shelf-life	≥ 36 months

Program Development

Capabilities

Nitto program development is a tailored approach to each project’s unique demands, including:

PhD subject matter experts comprised of in vitro and in vivo scientists, analytical experts, business development and quality assurance.

Successful proof of concept at small scale until successful delivery is achieved.

Goal to ensure that LNP product specifications align with regulatory guidelines and expectations.

As each project is unique, we work to develop a criteria driven data set at small scale, that once successful delivery is achieved, can be quickly scaled to larger, engineering and clinical scales to help our clients move to the clinic as rapidly as possible.

Development Expertise

IND Enabling Studies

Leveraging Nitto’s experience with U.S., European and Japanese regulatory authorities, we can perform or recommend studies required for moving to the IND stage of development.

Bringing Nitto’s unique philosophies and ‘tailored approach’ to collaborative partners on an ongoing basis to promote successful outcomes.

Providing technical information necessary for filing your IND.

Supporting partners as clinical studies progress by providing necessary technical information.

Nitto is different from contract service companies that are fee for service models. Consistent with Nitto’s mantra to innovate for customers is our belief that “Your success is our success.”