It may just be symbolic, but the two-year-old gene therapy Capsida Biotherapeutics built its manufacturing site in the shadow of pharma giant Amgen's headquarters, a wink to its greater ambitions.
Capsida's new 15,000-square-foot manufacturing site in Thousand Oaks will be used to engineer small viral particles to treat rare diseases like ALS and Friedreich's Ataxia, both of which severely impact mobility.
It's a rare move for a company that young; most startups developing therapeutics often partner with a contract manufacturing facility that makes drugs for multiple companies. But Capsida, already a fully-integrated company that works on research and development and preclinical studies, will be able to create therapeutics at a higher rate than if they were to contract.
"We've decided that, given our platform and what we feel like we can do with capsid engineering, the speed to market and the ability to get to clinic is such that if we have our own internal capabilities, we can move faster. We can be more agile," Capsida Chief Manufacturing Officer Rayne Waller said.
Photo by Keerthi Vedantam
Capsida's facility comes with two production suites where equipment can move flexibly (which will be useful as the company scales up, scales out or pivots), a sterile fill room to package drugs, and a large storage facility to preserve drugs in freezers. The facility will employ 40 to 50 people. Capsida is still building out a research space that will be anywhere from 20,000 to 25,000 square feet that will look into different use cases for capsid engineering — and the facility can support three to four clinical programs a year, allowing Capsida to test for a variety of use cases.
Capsida's facility is yet more proof of the impact local funding can have on keeping companies in L.A. The company announced in April it raised $140 million from Westlake Village BioPartners, a two-year-old venture capital firm founded by Amgen executives that raised one of the biggest funding rounds of 2020 in L.A and invested in several life science companies in Southern California. The firm recently built out an incubator space and office spaces for companies like Capsida to move into.
Venture capital firms like Westlake Village BioPartners are key to establishing biotech companies in L.A., according to Stephanie Hsieh, head of the Los Angeles' office for Biocom. In order to successfully pull off drug development, she said startups need need "the right investors with the sophistication and the knowledge of what it takes to really invest in biotech."
"It's super long lead times. It's a really complex product when you're talking about drugs and all that goes into it," Hsieh said.
The facility is another win for Thousand Oaks, which changed its zoning laws two years ago in a bid to accommodate more biotech companies. Capsida's facility is surrounded by medical technology facilities, including manufacturer ECA Medical Instruments, immunotherapy company Atara Biotherapeutics and pharma giant Amgen.
"We realized early on how important it is to make way for growth in the sector and to attract some of the best research companies in the world," said Thousand Oaks Mayor Claudia Bill-de la Peña.
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Llewellyn Cox is making a WeWork for bioscience professionals.
In a building along a hip arts district in Atwater Village, the molecular biologist and venture investor will open his next no-strings-attached lab space where he hopes startups can plant their roots in Los Angeles and help create therapeutics that save lives.
For $4,600 a month, a biotech startup can access a university-grade lab created by Lab Launch, which Cox founded in 2014. The company broke ground on the project Tuesday.
While the concept isn't new, there are few of these lab locations available in Los Angeles. Cox is part of a small group of life science professionals that are trying to bolster the region's biotech economy and prevent budding talent from straying to hubs in San Diego, Boston or San Francisco.
"The way to really build an economy is to start companies and grow companies and keep them here and hire people from here," Cox said. "And that was always a challenge when we were first starting."
Unlike tech upstarts that have humble beginnings in cramped garages or dingy basements, biotech startups need expensive lab equipment to store samples and dispose of biohazard disposal, not to mention cumbersome permitting. The upfront costs can be daunting and often deter would-be entrepreneurs, funneling them into larger companies or university incubators that might take over some patent ownership.
Cox's answer is Lab Launch.
"In the absence of all external incubators, you basically have to jump seat-first into the commercial real estate market while starting up a company, or try to sublease an industrial space in the middle of nowhere and build your own lab, and these are really hard things to do," Cox said.
Stephanie Hsieh runs one of the region's largest trade organizations for life science professionals, but when she first arrived in Los Angeles two decades ago, she couldn't even find a lab to lease for Meditope. The cancer therapeutics startup that she helmed needed a lab where it could store samples and experiment with new therapies. The options were limited, and she moved the company's development to San Diego where there was ample infrastructure in which to innovate.
"I joke that we innovated here and then it moved to San Diego or the Bay Area because there was no reason for it to stick here," said Hsieh, of who heads the Los Angeles' office for Biocom, a San Diego-based trade organization that represents more than 1,400 life science companies.
Los Angeles has thousands of square feet of lab space, much of it backed by universities, private companies and venture capital firms, adding up to thousands of square footage.
Westlake Village BioPartners recently built out a 30,000-square-foot lab space in its new incubator campus in Thousand Oaks for budding startups to use. UCLA's Magnify, a short-term lab space that houses startups, prioritizes companies in which UCLA has intellectual property.
But Lab Launch doesn't look for a stake in the company. These rentable spaces allow startups to maintain independence and control over their intellectual property. And Cox argues it gives the bioscience industry much needed infrastructure.
In 2019, the industry generated $44.2 billion in economic activity, according to a report by Biocom. And the county also received a fourth of all funding the National Institutes of Health allocated to California that year, more than any other county in the state.
"There is a significant opportunity with bioscience to expand L.A.'s innovation economy, nurture homegrown talent and contribute to the long-term economic health of the community and city at-large," said Los Angeles City Councilman Mark Ridley-Thomas, who has spearheaded efforts to build more facilities for the industry.
At the Atwater Crossing Arts and Innovation complex, not far from the Los Angeles River, Lab Launch's 11,000-square-foot space is under construction. It will provide six private lab units ranging from 800 to 3,500 square feet and can be further subdivided based on how many companies join. The labs are near a cluster of hospitals in Silver Lake like Children's Hospital and Kaiser Permanente, a metro stop and a slew of restaurants and coffee shops.
Created by scientists and entrepreneurs, Lab Launch's first space opened in seven years ago in Monrovia. The 11,000-square-foot lab space provides a place where startups can collaborate and experiment. The facility comes with ultra pure water (a common ingredient in the pharmaceutical industry) and freezers made to store samples.
The most common clients are small research and manufacturing organizations that contract with other, often larger, pharmaceutical or biotech companies and focus on specific niche verticals, like creating a singular pharma ingredient over and over again and only need a bit of lab space to do so. These organizations make the development and manufacturing part of biotech and pharmaceuticals cheaper.
KorvaLabs was one of the first companies at Lab Launch's Monrovia facility in 2015 to develop and test performance-enhancing drugs. The company stayed for two years before growing out of it and into another facility in San Dimas. It later worked with and was bought out by COVID-testing company Curative during the pandemic.
"Having these kind of established professionals around makes it a much more supportive and creative environment for those high growth startup companies that need help wherever they can get it," Cox said.
Hsieh already sees it happening.
"Just in the last five years, I'll say we've just seen explosive acceleration and growth," Hsieh said.
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A Thousand Oaks startup that is creating gene therapies aimed at permanently treating devastating diseases like Alzheimer's announced Thursday it received $140 million in capital.
Capsida Biotherapeutics is joining the gene therapy craze with small encapsulated viruses known as adeno-associated viruses, which can target specific cells in the body to treat diseases. The method is similar to the one used by pharma companies that create coronavirus vaccines.
Westlake Village BioPartners and Versant Ventures backed the Series A round with $50 million. Another $90 million came from pharma company AbbVie through a partnership.
Gene therapies aren't new — as a matter of fact, gene therapies have been heralded as a safer, more long-term solution to many diseases. Their approach is often try to replace or get rid of mutated genes that are causing harm to the body, or add genes to help the body fight diseases like Parkinson's or Huntington's.
"Unlike a pill that you take your once, twice a day, it's one treatment and you're done," said Robert Pacifici, chief scientific officer at a nonprofit Huntington's research organization called CHDI Foundation.
Several recent gene therapies have yet to break through. The FDA recently rejected the approval of BioMarin's gene therapy for hemophilia bleeding.
Others, like BlueBird Bio (which billed a promising sickle-cell gene therapy) and Ultragenyx (which was testing a gene therapy cure for Angelman syndrome) suspended clinical trials after patients had adverse effects.
There are often two problems to using gene therapy — targeted therapies don't always get to their destination, and, in large doses, often lead to negative effects, but in small doses, don't produce the desired results. It's a delicate balancing act for drug developers who need to be able to prove their therapy works without harming the body.
Capsida Biotherapeutics CEO Robert Cuddihy
(Courtesy of Capsida Biotherapeutics)
"There's a whole swath of areas that really haven't been amenable to any treatments," said Capsida CEO Robert Cuddihy. "I've always had an interest in the gene therapies and been following it for more than a decade, but it really wasn't ready for prime time."
Capsida uses — as its name would suggest — capsids, the outer coating of a viral particle that gets injected into the body. The capsid increases the particle's ability to hone in on a specific organ or cell, making targeting easier.
"The same amount of virus can actually do a whole lot more because it's much better getting into the cell," Cuddihy said. "It's much more effective. That then lets you decrease the dose," which lowers the risk profile of the virus.
The technology behind Capsida came from Caltech neuroscientists Viviana Gradinaru, who took existing capsids that could only transfer around 5% to 10% of the genetic material to the body and upped it to 70%, improving efficacy. She also engineered capsids to de-target the liver (which sucks in many gene therapies), making it easier for the body to send the capsid to the necessary cell.
Inside the capsid, where specific genes or therapies are located, the company will use AI and machine learning to help guide the development of gene therapies through the body and find its precise targets.
Capsida's partnership with AbbVie net the company $80 million in cash and $10 million in equity, with the potential of $530 million, as both companies make steps in targeting three specific diseases that deal with the central nervous system.
The technology could prove to be immensely useful for rare diseases like Huntington's, or prevalent, devastating diseases like Alzheimer's (though the company declined to mention which three diseases it would be targeting).
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