Nanite

Company Overview

Nanite is a next-generation non-viral gene delivery company developing a new class of programmable polymer nanoparticles for a range of modalities and indications. Founded in 2021 and based in Boston, Massachusetts, Nanite is pioneering the future of genetic drug delivery with safe and resorbable polymer nanoparticles (PNPs).

The company’s mission is to solve the biggest hurdle to gene therapy - delivery. Nanite aims to harness the power of AI for non-viral gene delivery, developing programmable, targeted, and safe polymer nanoparticles for a broad range of modalities and indications. Their vision is to redefine the boundaries of what’s possible in gene delivery.

Products Overview

Nanite’s core product is its AI-driven platform called SAYER. This platform combines cutting-edge high-throughput experimental and computational methods to design fit-for-purpose delivery vehicles delivering a broad range of genetic cargoes with tissue specificity.

The SAYER platform integrates:

1. High-throughput synthesis automation to generate chemically diverse polymers
2. Complexation of polymers with varied nucleic acid cargos to yield PNPs
3. Proprietary first-principle assays for meticulous characterization of PNPs
4. High-throughput in vitro screening for expression, endosomal escape, and toxicity
5. In vivo multiplexing to evaluate biodistribution, tropism, and cell specificity
6. AI-powered polymer design for high-accuracy predictions of PNP-cell interactions

The resulting polymer nanoparticles (PNPs) are designed to be:

• Resorbable and safe, reducing immune responses and enabling repeat dosing
• Tunable and modular, allowing rapid prototyping and adaptation for specific delivery demands
• Scalable in manufacturing, with potential for rapid production and cost-effectiveness
• Engineered for stability across various environmental, storage, and transport conditions

Founding Team

1. Sean Kevlahan, Co-founder and CEO: PhD in Chemical Engineering, previously co-founded Quad Technologies (acquired by Bio-Techne in 2018) and led Bio-Techne’s cell and gene therapy division through 2021.

2. Shashi Murthy, Co-founder and CTO: PhD in polymer science from MIT, co-founded Quad Technologies and founded Flaskworks (acquired by Northwest Bio).

3. Thomas Neenan, Co-founder and CBO: PhD in the chemistry of materials, co-founded Panbela Therapeutics (NASDAQ: PBLA) and AbFero Pharmaceuticals (acquired by Pharmacosmos).

4. Felipe Oviedo, Head of ML/AI: PhD from MIT, previously worked at Microsoft and CERN on AI applied to scientific and engineering problems.

Problem and Market Fit

Nanite is addressing a critical challenge in the field of gene therapy - the limitations of current delivery methods. Traditional viral vectors used in gene therapy face several issues, including limited payload capacity, potential immune responses, and manufacturing complexities. Nanite’s polymer nanoparticles aim to overcome these limitations by providing a safer, more flexible, and potentially more cost-effective alternative for delivering genetic therapies.

The company’s focus on non-viral delivery aligns with a growing trend in the gene therapy field. As noted in a Nature Biotechnology article from June 2023, companies are increasingly looking to replace viral vectors with new delivery technologies to overcome limitations and reduce production costs.

Business Model

While specific details of Nanite’s business model are not explicitly stated, the company appears to be pursuing a collaborative approach with potential partners in the biotechnology and pharmaceutical industries. This is evidenced by their strategic collaborations, such as the one announced with SalioGen Therapeutics for developing a lung-targeting nanoparticle for cystic fibrosis genetic medicine.

Nanite is likely to generate revenue through: 1. Research collaborations and partnerships with biotech and pharmaceutical companies 2. Licensing of their SAYER platform technology 3. Development of custom PNPs for specific therapeutic applications

Funding and Runway

In February 2023, Nanite raised $6 million in seed funding to launch its artificial intelligence platform for gene delivery. The company has also received grant funding, including:

• Up to $2 million investment from the Cystic Fibrosis Foundation to explore novel methods to deliver genetic therapies to the lung.
• $1.8 million award from the Bill & Melinda Gates Foundation to develop AI-driven gene therapy for HIV.

These funding sources suggest that Nanite has financial runway to continue its research and development efforts, though specific details about the company’s burn rate or projected runway are not provided.

Competitive Landscape

Nanite operates in the competitive field of gene therapy delivery. While the company doesn’t explicitly list its competitors, it’s likely competing with:

1. Companies developing other non-viral delivery methods for gene therapy
2. Established viral vector manufacturers
3. Other startups and research institutions working on novel delivery technologies for genetic medicines

Nanite’s competitive advantage appears to lie in its AI-driven approach to polymer design and its focus on creating tailored, tissue-specific delivery vehicles.

Customers

While specific customers are not listed, Nanite’s potential customers include:

1. Pharmaceutical and biotechnology companies developing gene therapies
2. Research institutions working on genetic medicines
3. Organizations focused on specific diseases that could benefit from gene therapy (e.g., Cystic Fibrosis Foundation)

Relevant News

1. May 29, 2024: Nanite announced a strategic collaboration with SalioGen Therapeutics to develop a non-viral, lung-targeting nanoparticle for cystic fibrosis genetic medicine.

2. May 8, 2024: The Bill & Melinda Gates Foundation awarded Nanite $1.8M to develop AI-driven gene therapy for HIV.

3. February 20, 2023: Nanite raised $6 million in seed funding to launch its artificial intelligence platform for gene delivery.

4. March 15, 2023: The Cystic Fibrosis Foundation announced an investment of up to $2 million to explore Nanite’s novel method to deliver genetic therapies to the lung.

These developments highlight Nanite’s progress in attracting both investment and strategic partnerships, indicating growing interest in their technology and approach to non-viral gene delivery.