Biotech startup Rampart Bioscience announced an $85 million Series A financing to continue its development of novel DNA-based medicines. The new financing builds on an initial $40 million seed investment from OrbiMed, bringing Rampart Bio’s total to $125 million in funding.
The La Jolla-based company’s proprietary DNA-based medicines platform is called HALO. It’s designed to produce potent, durable, and redosable therapies for patients with genetically driven diseases. Rampart Bio states that its science integrates capabilities from a variety of key scientific disciplines like gene delivery, protein sciences and clinical translation, allowing its therapies to surpass the limitations of current gene therapies.
Non-Viral Gene Delivery
For background, viruses are commonly viewed as effective forms of gene delivery – a virus can inject its DNA into host cells. The HALO platform, however, enables so-called non-viral DNA delivery, meaning it doesn’t use a virus to introduce genetic material into cells. These non-viral systems are generally considered safer and less toxic, but critics argue that they may be less effective, among other concerns.
“The non-viral space has gotten more recent attention as viral gene therapeutics have faced difficult and persistent challenges due to their inability to re-dose, small packaging capacity, problems with addressing inadequate initial response or declining expression, high-dose safety issues and price,” said Rampart Bio CEO and Co-founder Louis Breton. “We feel privileged that our early in vivo proof of concept work that highlighted the potential for potent, durable, re-dosable, titratable and safe gene medicines helped us to raise this important round.”
Rampart Bio Co-founder and Chief Innovation and Technical Officer Dr. Jeffrey Bartlett, PhD added, “We believe this discovery holds the key to overcoming the significant potency and durability challenges still facing predecessor approaches that are capable of reaching certain therapeutic cell types but are not eliciting robust or durable therapeutic effect.”
Breton and Dr. Bartlett previously worked together at gene and cell therapy company Calimmune, which Breton co-founded and later sold to CSL Behring.
Treating A Rare Genetic Disease
Rampart Bio’s main target is the treatment of hypophosphatasia (HPP), a rare, often fatal genetic disease that prevents bone and teeth mineralization. Symptoms include skeletal deformities, bowed legs, enlarged wrists or ankle joints, bone and joint pain, frequent fractures and fatigue. According to a 2020 Mayo Clinic report, to date, more than 400 distinct gene mutations have been found in patients with HPP. Every 1 in 100,000 people develop severe cases.
“A number of our founding members worked on the only approved therapy for HPP, called Strensiq. Strensiq is an enzyme replacement therapy that treats approximately 30% of the most severe HPP patients, requires thrice-weekly dosing, and is extremely expensive,” he shared. “Our interest in utilizing our core technology to first address this patient population, that the team was intimate with, was a critical driver in establishing the company. The platform we developed, however, is not limited to this indication.”
Rampart says it’s too soon to comment on a commercial timeline for its HALO delivery system but reports it is making strong progress on its discovery and preclinical-stage programs.
FOUNDER & CEO: Louis Breton
HEADQUARTERS: La Jolla
FUNDING: $125 million (Seed and Series A)
NOTABLE: Rampart’s executive management team collectively has more than seven decades of biotech experience, including the development of gene and cell-based therapies.