Circumventing the need for viral vectors with a purely physical, decentralized solution for producing cell therapies.

CEO: Steven Banerjee
Based: San Francisco, California
Founded: 2017

The scoop: To deliver therapeutic pieces of DNA or genetic engineering inside cells where they can do their work, we largely rely on modified viruses to ferry them across membranes using the same molecular methods that viruses have evolved over ages to spread infections and disease.

But while gene and cell therapies offer great potential for leaps forward in treatment, with dozens more expected to be approved in the coming years, they come with equally high price tags—and the use of complex viral vectors for their delivery has helped balloon the costs of a single dose into the hundreds of thousands of dollars per patient.

Mekonos aims to sidestep these biological processes entirely, and use silicon nanoneedles to deliver therapeutic payloads directly into the cell in a purely physical, mechanical form factor—with the goals of greatly lowering the cost and time of development, avoiding manufacturing bottlenecks and perhaps enabling personalized doses to be produced in a hospital setting.

What makes Mekonos fierce: Picture an assembly line—millions of individual cells taken from a donor, all moving in parallel through a massive system, and sandwiched between two chips. Below, a microfluidic cell trap holds each one in place, while, from above, individually controlled needles dip into each cell to deliver the desired genetic payload. Each silicon needle is no more than 50 nanometers in size—about the width of a single Ebola virus—and can be coated with strands of DNA, RNA or CRISPR-Cas complexes.

“We see ourselves as developing elegant machines to save lives,” says Mekonos’ founder and CEO, Steven Banerjee, who describes the San Francisco and Silicon Valley-based firm as a technology company first, aimed at the backend infrastructure behind the design, development and production of gene and cell therapies. “We’re like Fedex, delivering the package at the right place and the right time.”

The company expects to be able to transport payloads into cells and nuclei without the restrictions of viral-based vectors, such as the length of nucleotide strands. While the commonly used adeno-associated virus vector can only hold fewer than about 5,000 base pairs of genetic information, Mekonos’ platform has delivered materials five times as large, Banerjee said. In addition, the system can be used to multiplex different materials into the cell at once, while maintaining viability rates north of 85%.

“These membranes are very robust; they can self-repair themselves.” Banerjee says, describing how the company has punctured single cells multiple times. “It’s a very precise, finely tuned microinjection at the end of the day.”

Currently, Mekonos’ test chips, produced at the Lawrence Berkeley National Laboratory, house between 800 to 1000 needles on a centimeter-by-centimeter square. The company hopes to scale that up to units containing hundreds of thousands to millions of needles over the next few years. The final product will resemble a modular stack of cartridges and hardware similar, to computer server blades found in data center racks.

Replacing the use of viral vectors wouldn’t only save gene and cell therapy manufacturers money, but time, by aiming to re-engineer millions of cells locally and within a 24-hour timeframe. By comparison, acquiring highly sought-after, clinical-grade viruses, which can be custom designed for each product, may take months or years to procure. It would also eliminate investments in the surrounding quality control protocols, used to check for cross-contamination risks and so on.

Mekonos’ major benefactor so far, Novartis, has provided the company seed funding as well as office and laboratory space through its Biome incubator program for digital health companies.

While Novartis’ pharmaceutical division hasn’t joined on as a strategic partner yet, the potential benefits should be obvious: the Swiss drugmaker’s Kymriah CAR-T cancer treatment was approved in 2017 with a famous $475,000 list price.

But that could be dwarfed by the company’s upcoming Zolgensma gene therapy, being developed by its AveXis subsidiary in spinal muscular atrophy—with a one-time dose costing between $4 million and $5 million. Last December, Novartis said it was weighing new insurance methods as a way to secure access.

Previously known as AVXS-101, Zolgensma employs an AAV vector to deliver a replacement copy of a transgene that codes for specific motor neuron proteins. It’s currently under priority review at the FDA, with a decision expected by the end of May.