The buzzy biotech Verve is gearing up to test a gene-editing treatment that could cure heart disease. The CEO shares his 3-step vision for the one-and-done heart treatment.

Verve Therapeutics cofounder and CEO Sekar Kathiresan

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A Cambridge, Massachusetts biotech chasing the goal of curing heart disease just announced the first treatment it plans to test in people.

Verve Therapeutics’ lead experimental therapy, dubbed VERVE-101, aims to slash bad cholesterol levels and effectively cure patients with a serious genetic heart disease by editing a tiny piece of their DNA. Verve CEO and cofounder Dr. Sekar Kathiresan told Business Insider that’s the first part of his three-step strategy in transforming how heart disease is treated.

“Ultimately, this medicine, if effective and safe, will be a one-and-done for heart attack,” Kathiresan said.

Verve is set to be one of the leading — if not the first — biotechs to begin testing cutting-edge gene-editing technologies in humans to treat heart disease. Other biotechs have started testing CRISPR-based treatments for the genetic blood disorders sickle cell disease and beta-thalassemia.

Kathiresan, a prominent cardiologist, founded Verve while working as a researcher at the Broad Institute and Massachusetts General Hospital. After a lifetime of studying the heart and its related genetics, Kathiresan resigned from his positions to lead Verve, which was incubated by GV, formerly known as Google Ventures, from 2016 to 2018. 

Over the last two years, Verve has raised more than $120 million in seed and Series A rounds led by GV. The company now has 53 employees, with plans to grow to about 80 workers in the next two years, Kathiresan said. Most of the hiring will be for clinical research and manufacturing roles, he added. 

New data shows sustained cholesterol drop in monkeys, further building enthusiasm for gene-editing research

The biotech’s first experimental treatment was built using tools from the biotech Beam Therapeutics called base editing technology. It’s a more precise way to edit genes, allowing scientists to change a single letter of genetic code.

In this case, VERVE-101 aims to change an A to a G in the code of a gene involved in regulating cholesterol levels, called PCSK9. That single infusion should permanently lower someone’s bad cholesterol levels. 

Verve’s first study will enroll patients with heterozygous familial hypercholesterolemia, a genetic heart disease that affects about 1.6 million people in the US and Europe, Kathiresan estimated.

These patients have abnormally high levels of LDL, or bad cholesterol from birth, which often result in overworked arteries. The ultimate result is young patients, often in their 30s or 40s, having heart attacks, strokes, and sometimes even dying. 

The technology isn’t ready to be tested in people. Verve will focus in 2021 on completing the necessary toxicology studies and lab work to set the stage for human testing to begin in 2022, Kathiresan said.

Even as the technology remains a ways away from potentially reaching large numbers of patients, excitement is clearly building in the space.

Verve’s partner Beam, for instance, went public in February 2020 at a valuation of more than $1 billion. Less than a year later, Beam’s stock has increased more than 350%, with the biotech now worth well over $5 billion, despite having no treatments ready for testing in people.

Verve’s latest monkey data, released Tuesday morning, should further bolster enthusiasm. Initial data, presented in June 2020, showed the technology could effectively block genes to dramatically lower cholesterol levels in monkeys.

“This could be the cure for heart disease,” Dr. Michael Davidson, a cardiovascular surgeon not involved in the research, told The New York Times.

In Tuesday’s update, Verve showed that cholesterol drop of 61% was sustained six months out and counting, based on results from four monkeys. These are the first monkey studies to show a prolonged benefit from the base-editing technology, Kathiresan said.

The long-term goal is to develop a one-and-done treatment that can prevent heart attacks

The company’s long-term vision is to tackle cardiovascular disease, the leading cause of death in the US. The initial work in patients with this genetic heart condition is the first of three steps, Kathiresan said.

“Step two and step three are even more exciting,” he added. 

The second phase will expand research to anybody with atherosclerotic heart disease, a group that Kathiresan estimated at about 24 million people in the US and Europe. That’s a condition in which plaque builds up in arteries, narrowing them and potentially blocking blood flow or causing them to break.

The third step is the most ambitious: testing a gene-editing program as a preventive therapy for anyone at risk of heart attack. This would require a sizable amount of safety data in humans, Kathiresan acknowledged — data that doesn’t yet exist at any level for base-editing treatments. 

In chasing that ambitious vision, Verve will soon need to raise more capital. Kathiresan declined to say if Verve plans take advantage of a red-hot IPO market and go public this year.

“We’re in the process of thinking through what those additional raises are going to be like,” he said. “That’s probably all I want to say right now on that topic.”

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One of biotech's most valuable startups just filed to go public. Here are 5 crucial takeaways from Sana's 271-page filing.

Summary List PlacementAfter raising more than $700 million since its 2018 launch, one of the biotech industry’s most valuable private companies is planning to go public.
Seattle-based Sana Biotechnology filed paperwork on Wednesday for an initial public offering. Following a red-hot year for biotech IPOs in 2020, Sana is hoping to take advantage of investor enthusiasm to fuel its ambitious cell and gene therapy programs.
The company was founded in July 2018 and is led by former executives of Juno Therapeutics, a cell therapy startup that was acquired in 2018 by Celgene for $9 billion. Sana came out of stealth mode in early 2019 and closed a Series B round in June 2020 in which it raised $435.6 million.
The size of its latest funding round places Sana among the top 10 biotech or life sciences companies in the US, according to data going back to 2002 from PitchBook. Some of the other companies on that shortlist include coronavirus-vaccine-maker Moderna, low-cost drugs startup EQRx, cancer-detection company Grail, and disgraced lab-testing firm Theranos.
Read more: The 26 billion-dollar startups to watch that are revolutionizing healthcare in 2021
Sana commanded a $2.77 billion valuation after the 2020 raise, the second-highest valuation among all private biotechs, according to Silicon Valley Bank’s annual industry report. Sana hasn’t set yet set the pricing terms for its IPO.
The company plans to trade on the Nasdaq under the ticker symbol SANA. Morgan Stanley, Goldman Sachs, JPMorgan, and Bank of America are leading the offering.
We read through Sana’s 271-page filing to learn more about the secretive biotech’s business and strategy. Here are five crucial takeaways from Sana’s latest disclosure. 
Sana’s pipeline is sprawling, but it’s at least a year away from starting human testing
Sana’s filing provides the first detailed look at the treatments it’s working on, and it’s sprawling in terms of both its scientific ambitions and the range of disease areas it’s targeting. 
The biotech’s lead program is focused on a rare genetic blood disorder called ornithine transcarbamylase (OTC) deficiency. Beyond that, the company has early-stage treatment candidates in development for heart failure, Type 1 diabetes, multiple sclerosis, Huntington’s disease, several types of cancer, sickle cell disease, and beta-thalassemia.
Sana’s platform is primarily focused on engineering and manipulating cells. But it’s also researching gene delivery, gene modifications, and pluripotent stem cells.
All this work is still in the earliest stages of development. None of its potential treatments are being tested in people, and Sana doesn’t expect that to happen until 2022 at the earliest.
Biotech companies deciding to go public before any of their treatments are tested in people has been a trend in the last few years, as investor appetite for early scientific bets has grown. By that count, Sana is shaping up to be a mammoth wager. 
The startup has set up an independent research arm and is digging into COVID-19
Sana’s S-1 also gives details on the company’s research arm SanaX, led by Harvard Medical School genetics professor Richard Mulligan. The group is currently investigating new tools for Sana’s cell and gene therapies, including developing new viral vectors, expanding the capacity of existing vectors and manipulating the body’s immune response. 
On top of that, SanaX is exploring how to best delivery specific antibodies to treat the novel coronavirus. That could put the startup in competition with industry giant Regeneron, which has teamed up with gene therapy pioneer Jim Wilson to find new ways of delivering antibodies to fight the virus. 
New tools that come out of SanaX could be folded into Sana’s internal drug portfolio or be used in an external partnership. The company has yet to announce any drug industry collaborations.
“Our goal is to lead both the present and future of cell engineering and we are committed to making significant investments in research and other activities that will ensure a leadership position throughout the next decade,” the S-1 said. 
ARCH and Flagship own nearly half of Sana’s shares
ARCH Venture Partners and Flagship Pioneering are the main venture capital firms backing Sana. 
ARCH owns 27.5% of Sana’s stock going into the offering, and Flagship owns 21.4%. The next largest institutional investor is Canada Pension Plan’s investment fund, which owns 5.8% of the biotech.
How much those shares will ultimately be worth is an open question, as Sana has yet to specify its target pricing range. Most biotech IPOs offered shares somewhere around $15 to $20, which would effectively value Sana at somewhere around $9 billion to $12 billion, Stat News’s Kate Sheridan reported.
Judging by its $705.5 million in previous funding, Sana isn’t a typical biotech. If Sana prices at $20 per share, ARCH’s and Flagship’s stakes would respectively be worth $3.5 billion and $2.7 billion. 
The biotech hasn’t built its own manufacturing presence, instead relying on third parties
The drugs Sana aspires to make are very complex. The filing reveals that Sana hasn’t spent its copious funding on building its own manufacturing capacity. 
“We do not yet own or operate any cGMP manufacturing facilities,” the filing states, referencing current good manufacturing practices, the universal standards enforced by drug regulators. 
Instead, like many pre-commercial biotechs, Sana is relying on third-party contractors to produce its experimental treatments.
While the company doesn’t have a manufacturing footprint, it has built up a nationwide research presence with about 170,000 square-feet in office and lab space across Seattle, South San Francisco, and Cambridge, Massachusetts. 
Sana has been hungry for growth, executing three acquisitions worth a combined $1.5 billion
Sana has made several acquisitions in its first two years of operations, which combined are worth more than the funding it has raised to date. The company has mainly used its own stock to fund the deals.
Its largest was the February 2019 acquisition of Cobalt Biomedicine, which was also incubated at Flagship Pioneering but was still in stealth mode when it was acquired. Sana paid $136 million in stock in the deal, which included the possibility of $500 million in milestone payments and a $500 million “success payment” tied to its valuation.
The acquisition gave Sana access to the in-vivo gene therapy approach used in its lead drug candidate. It also helped Sana grow its workforce, which has ballooned from 37 employees at its launch to more than 200 employees currently. 
Sana has further built out its broad pipeline through the acquisition of two Seattle-based startups. It bought  Cytocardia Inc. in November 2019 for upwards of $148 million and acquired Oscine Corp. in September 2020 for upwards of $234.3 million. Join the conversation about this story » NOW WATCH: Sarah McBride made history becoming the first openly trans person elected to a state Senate seat. In 2018, she explained why the Trump administration wouldn’t discourage her work.

THE RISE OF GENETIC TESTING IN HEALTHCARE: How leading genetic testing companies like Ancestry and 23andMe are carving into healthcare with the promise to fuel more personalized care

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Insider Intelligence publishes thousands of research reports, charts, and forecasts on the Digital Health industry. You can learn more about becoming a client here.
The following is a preview of one Digital Health report, The Genetic Testing in Healthcare Report. You can purchase this report here.

Since the first human genome was sequenced in 2003, the genetic testing market has evolved rapidly alongside consumers’ interest in how their genetic makeup affects their health.
The human genome was sequenced — or read in its entirety — for the first time in 2003 after more than 20 years of work and nearly $5 billion was put into the National Institutes of Health’s (NIH’s) Human Genome Project — which marked a huge step in helping scientists and medical researchers understand how genes and gene interactions impact disease development and progression. In the ensuing decades, genetic information catapulted into mainstream healthcare, driven largely by the rapid decline in cost for DNA sequencing technology.DNA testing firms like Ancestry and 23andMe broke onto the genetic testing scene via direct-to-consumer (DTC) tests, and consumers have flocked to their products seeking to gain insights into their individual health risks. Ancestry and 23andMe both offer cheaper, but less comprehensive, DNA testing — their products come at a price point between $100-$200, which is likely more enticing to average consumers than higher-cost tests that explore the entire, or a larger portion of, the genome.
Of the 26 million global consumers who took a DNA test in 2019, Ancestry and 23andMe tested 25 million. This marks a meteoric rise in consumer adoption over the course of the 2010s: In 2015, for example, fewer than 1.5 million global consumers had taken at-home genetic tests. 
The ability to provide genetic tests at a lower cost has opened up new opportunities in preventative medicine. For example, chronic illness accounts for 90% of the US’ more than $3.7 trillion of annual healthcare spending — and healthcare stakeholders are actively looking for ways to assess population health risks and intervene earlier. Genetic testing is an attractive proposition for healthcare players that want to paint a picture of an individual patient’s health risks — and use that information to help guide care plans that could mitigate the development or progression of a condition and steer drug development for more precise medications.
In this report, Insider Intelligence will examine the industry forces that have helped evolve genomic information from a consumer novelty to a transformative healthcare technology. We outline how some of the key players in the genetic testing space have altered their business models to appeal not only to consumers but also healthcare players across the industry, including health systems and pharma companies. We provide a glimpse at what’s next for the implementation of genomic information — namely, the barriers that are holding genetic testing companies back from reaching new customers, including how the coronavirus pandemic could impact growth in the space.
The companies mentioned in this report are: 23andMe, Almirall, Ancestry, Apple, Calico, Color, Diploid, Genelex, GlaxoSmithKline, Google, Helix, Illumina, Invitae, Jungla, LunaPBC, Mayo Clinic, Microsoft, MyHeritage, NorthShore University Hospital , Novartis, Ochsner Health, Pfizer, PWNHealth, Salesforce, Stanford Medicine, TrialSpark, Verily, Veritas, and YouScript.
Here are some key takeaways from this report: 

The use of at-home, direct-to-consumer genetic tests skyrocketed during the 2010s, but has been tapering off over the last couple of years. 
The slowdown in the DTC market has catalyzed genetic testing companies to veer into healthcare — teaming up to give legacy healthcare players access to rich sets of data that could guide care and treatments, which could help boost customer bases. 
Health systems can add genetic testing into care regimens to gain a more comprehensive image of patients’ health risks. Payers can front the costs of tests for their members, the results of which could empower them to take proactive approach to care management and reduce costs in the long-run; and drug-makers can unlock troves of genetic data in order to design more precise medications.
Myriad barriers are still holding back genetic testing companies from cementing their position in the healthcare industry, including privacy and accuracy concerns, the inability of healthcare professionals to operationalize genetic data, and a continued drop in consumer interest. 
The coronavirus pandemic and accompanied economic slowdown could weigh on genetic testing companies further, as consumers will likely put off nonessential purchases like genetic tests, and healthcare companies may focus resources and time on initiatives more directly related to the virus and recouping lost revenue.

In full, the report: 

Provides an overview of the ways in which prominent US genetic testing companies are using their products to edge deeper into the healthcare landscape.
Highlights how leading telehealth vendors have reacted to a sudden, rapid uptick in adoption of their services.
Outlines how legacy healthcare players — including health systems, payers, and drug-makers — are leveraging genetic testing companies’ products to make use of insights into genetic variants.
Identifies the barriers that genetic companies are staring down that could hinder growth and adoption. 

Interested in getting the full report? Here’s how you can gain access:

Join other Insider Intelligence clients who receive this report, along with thousands of other Digital Health forecasts, briefings, charts, and research reports to their inboxes. > > Become a Client
Purchase the individual report from our store. > > Buy The Report Here

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