An Orbimed-backed biotech launches with $85M and plans for a new kind of DNA medicine
2023-10-24
基因疗法
When developing new medicines, drugmakers typically try to make treatments that are more effective, durable or safer than what’s already available.
If they’re lucky, they can hit on something that meets at least one of those goals. Rampart Bioscience, a new biotechnology company eyeing a way to make better DNA-based medicines, is striving to hit all three.
Headquartered in La Jolla, California, Rampart emerged from stealth Tuesday with $85 million in new funding to develop its lead program for a rare inherited condition known as hypophosphatasia.
The company is led by Louis Breton and Jeffrey Bartlett, a pair of biotech veterans who last oversaw Calimmune, a cell and gene therapy developer that worked to create new treatments for HIV and other diseases. Calimmune was picked up by CSL Behring in 2017 through a $91 million deal.
Their latest endeavor was born after a phone call with David Bonita, a partner with the venture capital fund OrbiMed.
OrbiMed previously funded another company developing a treatment for infants whose bones and teeth did not mineralize properly, which would later become Alexion Pharmaceuticals’ enzyme replacement therapy Strensiq. But the drug is used for severe forms of hypophosphatasia, leaving patients with more moderate symptoms without any options approved by the Food and Drug Administration. Breton and Bartlett wondered: Could they do better?
Rampart, which takes its name from a word meaning “to fortify,” hopes to create a treatment that could be more long-lasting and effective for those patients. The severe form of hypophosphatasia affects an estimated 1 in 100,000 babies, but milder cases likely occur more frequently and later in life.
“Especially for those who have diseases where it impacts their quality of life, helping them fortify their own bodies ... is a great step,” Breton said.
Genetic medicines have, especially in the last decade, proven they can dramatically benefit patients with certain inherited conditions. Often, though, their effectiveness has been hampered by limitations of delivery. Rampart believes its approach can address both delivery and the issue of re-dosability, which may be necessary as hypophosphatasia progresses, according to the company.
“There are many groups that have been working for specific rare diseases on genetic therapies that potentially would be a one-and-done. But in the way of looking at what would be optimal for patients, this provides more flexibility for doctors,” Breton said. “It's not just the availability of options for patients; it provides much more specificity to their symptoms and some of the aspects of their own disease progression.”
Rampart is also developing an unspecified number of other programs.
“We are very much a product company,” Bartlett said. But Rampart's near-term milestones also include “continuing to develop the platform and collect the kind of data we've been fortunate in getting.”
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