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COVID-19 has ravaged the economy, and it was expected to quash the IPO market, too. But the biotech sector is defying the pandemic with crossover financings and freshly minted public companies. On Friday, three firms added their names to the list of life science companies preparing to join the public markets.

Gene therapy company Generation Bio, vaccines developer Vaxcyte, and cancer diagnostics maker Burning Rock Biotech each filed IPO paperwork just ahead of the Memorial Day weekend. The filings come as an index of the largest and most liquid IPOs of the past two years reached an all-time high, according to Renaissance Capital. The IPO research firm says the indexs rise was led by Moderna (NASDAQ: MRNA), the Cambridge, MA-based biotech that this week released preliminary Phase 1 data for its experimental COVID-19 vaccine.

Investors are betting that new technologies and services are best suited for the post-pandemic world, Renaissance says.

Heres a look at the three new additions to the biotech IPO queue.

GENERATION BIO EYES NEXT-GEN GENE THERAPIES

Generation Bio aims to improve upon gene therapy with an alternative to the engineered viruses currently used to ferry these therapies into cells. Viral delivery has limitations that include safety risks and a relatively small genetic payload capacity, the Cambridge-based company says in its filing. Furthermore, if patients dont already have antibodies to the viruses, they develop them after their first dose, which means patients cant receive additional doses if the initial one doesnt work as expected or stops working over time. Gene therapies that employ viral delivery are also expensive to manufacture.

Instead of a virus as its delivery vehicle, Generation Bio uses a lipid nanoparticle (LNP). The LNP encapsulates its genetic payload, a DNA construct called closed-ended DNA. This approach permits an individualized approach to treatment as a patient can be redosed until reaching the level needed for effective treatment, the company says. The technology also has a greater payload capacity and its less expensive to manufacture at scale compared to viral gene therapies. Those differences will enable delivery of gene therapies to more types of tissue, which in turn will allow for the treatment of a broader range of diseases spanning more patients, Generation Bio says.

Generation Bios initial focus is developing gene therapies targeting diseases of the liver and the eye. The most advanced liver programs are for phenylketonuria (PKU), an inherited metabolic disorder, and hemophilia type A, the most common form of the bleeding disorder. For the eye, Generation Bio is developing a gene therapy for an inherited form of vision loss called Leber congenital amaurosis 10 (LCA10) and for Stargardt disease, which is a form of macular degeneration.

Those programs trail other experimental gene therapies that are currently in clinical testing. Pfizer (NYSE: PFE) is developing a hemophila A gene therapy that came from the labs of partner Sangamo Therapeutics (NASDAQ: SGMO). BioMarin Pharmaceutical (NASDASQ: BMRN) is testing gene therapies for hemophilia A and PKU. In LCA10,Editas Medicine (NASDAQ: EDIT) is developing a gene therapy under a partnership with Allergan, now a subsidiary of AbbVie (NYSE: ABBV). That therapeutic candidate uses the CRISPR gene-editing technology to correct the faulty gene inside the patient. All of these experimental gene therapies use adeno-associated viruses to reach their targets.

Generation Bio has raised more than $227 million, most recently a $110 million Series C financing in January. That funding round added crossover investors, whose involvement is viewed as an indication a company is preparing for an IPO. CEO Geoff McDonough acknowledged as much at the time, telling Xconomy he expected to take the company public in advance of beginning clinical trials. Other rare diseases that the company is exploring include Wilsons disease and Gaucher disease.

In its filing, Generation Bio set a preliminary $125 million target for its IPO. The company has applied for a Nasdaq listing under the stock symbol GBIO. At the end of the first quarter of this year, Generation Bio reported having $104.5 million in cash. The company says it plans to use the IPO proceeds to continue R&D, including the preclinical work to support an application to start clinical testing of one of its liver disease gene therapies.

Generation Bios largest shareholders are Jason Rhodes, the companys chairman and founding CEO, and Atlas Venture. Each holds a 37 percent pre-IPO stake, according to the filing. Fidelity Investment owns 14.9 percent of the company, followed by funds advised by T. Rowe Price, which hold 8.9 percent.

VAXCYTE SETS SIGHTS ON TOPPING A PFIZER VACCINE

Vaxcyte is the new name for SutroVax, which changed its moniker this week. The Foster City, CA-based company spun out of Sutro Biopharma, and it develops vaccines using technology licensed from its former parent. The company says in its IPO filing that its cell-free protein synthesis technology enables it to design protein carriers and antigensa vaccines key componentsthat are better than what can be produced using conventional vaccine technologies.

Pneumococcal bacteria, which can cause pneumonia and meningitis, are Vaxcytes first target. The top pneumococcal vaccine, a Pfizer (NYSE: PFE) product called Prevnar 13, is a blockbuster seller that protects against 13 of the more than 90 pneumococcal strains. Vaxcytes preclinical vaccine candidate, VAX-24, is being developed to address 24 strains.

The IPO filing comes two months after Vaxyte closed a $110 million Series D round that added crossover investors. The company says in the filing that it has raised about $282 million cumulatively. As of March 31, Vaxcytes cash holdings totaled $154.7 million. The companys largest shareholders include Abingworth Bioventures, Longitude Capial Management, and Roche Finance, though the percentages of those stakes were not disclosed.

Vaxcyte says it plans to apply for a Nasdaq listing under the stock symbol PCVX. The vaccine developer set a preliminary $100 million goal; proceeds will be used to complete preclinical development and advance VAX-24 into human testing. The cash will also finance manufacturing, as well as continued development of other vaccine candidates.

BURNING ROCK BIOTECH BLAZES A PATH TO NASDAQ

Burning Rock Biotech is based in China, where it sells next-generation sequencing products that help physicians select cancer treatments for their patients. Now its seeking a Nasdaq listing that will give US investors a chance to grab a stake.

The company says in its filing that it offers 13 tests spanning solid tumors including cancers of the lung, prostate, and breast, as well as blood cancers. In addition helping physicians treat cancer patients, Burning Rock says its products support clinical trials conducted by large pharmaceutical companies, including AstraZeneca (NYSE: AZN), Bayer, and Johnson & Johnson (NYSE: JNJ). The companys central laboratory processes biopsy samples from hospital patients as well as from its pharmaceutical partners. The central lab business is the companys largest business segment.

Burning Rock reported $53.9 million in 2019 revenue. For the first quarter of 2020, revenue was $9.5 million. The company set a preliminary $100 million goal for its IPO, and says it plans to apply for a Nasdaq listing under the stock symbol BNR. According to the filing, Burning Rock expects to use the IPO cash for research and development of early cancer detection technologies, as well as for seeking approvals in China for additional cancer therapy selection products.

Image: iStock/peterschreiber.media

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan@xconomy.com.

Originally posted here:

Generation Bio Leads a Trio of Biotech Companies Aiming for the Nasdaq - Xconomy