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First in Human: Early-stage nanotechnology poised for 'inflection point'


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First in Human: Early-stage nanotechnology poised for 'inflection point'

Editor's note: We reinstated publication of this feature on June 17 after a temporary suspension to ensure robust breaking news coverage on the healthcare sector for our clients.

This is a recurring column on clinical research in the early stages of development, what is referred to as phase 1. These are treatments being used for the first time in a small number of human patients to determine safety, dosing and general pharmacological activity.

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Scientists say nanotechnology will lead to many new medicines in the coming years, but navigating these new discoveries through the early stages of research can be difficult.

Nanotechnology is defined by the U.S. Food and Drug Administration as any product that contains material of one to 100 nanometers in size. Put into perspective, a strand of hair is about 100,000 nanometers wide.

One of the leaders in the field of biological nanotech engineering is Massachusetts Institute of Technology professor Robert Langer, who has helped found about 40 companies based on technology created and developed in his Langer Lab.

When working on this new technology, such as nanoparticles designed to deliver medicine to certain cells inside a patient, Langer told S&P Global Market Intelligence that it is important to look at the big picture, including failures as well as successes.

"As with almost every medical technology I've seen, there are lots of ups and downs, and probably more downs than ups," Langer said. "At the end of the day, I think there's been great progress in the biomedical engineering field."

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Spherical nucleic acids developed by Exicure are designed to deliver genetic material to cells to treat various diseases in oncology, neurology and dermatology.
Source: Exicure

One of the first genetic nanotechnology products to hit the market in 2018 was Alnylam Pharmaceuticals Inc.'s Onpattro, which uses a technique known as RNA interference to treat a rare disease called hereditary transthyretin-mediated amyloidosis.

There had been a long journey to get to that point, as nanotechnology in particular comes with many unknowns, such as molecule stability and patient toxicity, Langer said.

Exicure Inc., a Skokie, Ill.-based biotech, is running several early- to mid-stage trials using its nanotechnology, called spherical nucleic acids, CEO David Giljohann said in an interview. Exicure focuses on the fields of immuno-oncology, neurology and dermatology.

"The challenge really has been taking those structures and coming up with versions that are scalable, manufacturable, stable and ultimately able to get out into the patient populations," Giljohann said.

Nanotech on the horizon

Both Langer and Giljohann pointed to areas where nanotechnology is poised to make a difference in the medical community, from new delivery mechanisms that can make vaccines easier to administer in the developing world to improved diagnostics and drug discovery methods.

"I think you're going to see a huge influx of drugs into late-stage clinical trials, looking at what's coming down the pipeline now from various different nanoparticle delivery formulations," Giljohann said.

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Massachusetts Institute of Technology Institute Professor Robert Langer

Source: Langer Lab

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Exicure CEO David Giljohann
Source: Exicur

One of the most pressing global healthcare challenges in 2020 is the coronavirus outbreak and Moderna Inc., one of the companies Langer helped found, is on the front line of vaccine development for this new biological threat.

Moderna's nanoparticle-driven science uses genetic engineering to trigger cells to create proteins that prevent certain infections. Its vaccines for Zika virus and influenza have already progressed to early clinical stages, while its COVID-19 vaccine is still in preclinical trials.

Although nanotech has come a long way, targeting drugs is far from straightforward.

"When it comes to nanotechnology in drug delivery, I think what people want to do is target cells better and that's been difficult," Langer said. "Nonetheless, nanotechnology has played a big role."

An inflection point

The use of nanotechnology in medical settings is spreading, but challenges remain.

"A couple of years ago, I would've said that I expected it to move faster from laboratory into clinical trials," Giljohann said.

Exicure has scaled three drugs into clinical trials and that transition took about four years.

"A lot of the early work has been done by pioneers in the space to solve some of the fundamental issues with those scaling and manufacturing issues and now we're finally reaching an important inflection point for the nanotech space and translating it to new medicines," he said.

Before going public in 2019, Exicure raised funds from investors such as Microsoft Corp. co-founder Bill Gates. Finding backers with a distant enough outlook is important to an early-stage company, Giljohann said.

"Folks like Bill Gates and others that have been involved along the way, they've been involved because they're interested in the five-, 10-, 20-year horizon," Giljohann said.