A new biotech company, Entrada Therapeutics, has launched with a $59 million Series A funding, which it says it will use to advance its multiple programs, including the treating of a rare and fatal mitochondrial disease.
5AM Ventures and MPM Capital led the financing round, with Roche Venture Fund, MRL Ventures Fund (MRLV) and Agent Capital also participating.
Dipal Doshi, who was announced as the president and chief executive officer of Entrada Therapeutics, said the biotech was “well-positioned to advance multiple programs through the application of our proprietary intracellular biologics platform.”
“Entrada has an opportunity to create the next generation of therapeutics for previously undruggable intracellular targets. Leveraging the exciting science from the lab of our co-founder, Dehua Pei, at the Ohio State University, we look forward to building a world-class team that will translate this platform into meaningful therapies,” Doshi said.
The Boston based company’s intracellular biologics platform is based on the work of its co-founder Dehua Pei of Ohio State University in cell-penetrating peptides. This approach “has been shown to deliver a variety of modalities into the cytosol of the cell … including small molecules and large nucleic acids aimed at previously undruggable targets.”
In the past, Pei has looked into therapeutic areas such as inflammatory and autoimmune diseases, cancer, cystic fibrosis, Type 2 diabetes and organ transplantation.
In a statement, Entrada, which was funded and incubated within 5AM’s 4:59 Initiative via a seed investment by 5AM Ventures, Roche Venture Fund and MRLV, said its proprietary intracellular biologics platform has the demonstrated ability to deliver a variety of modalities into the cytosol of the cell, allowing for the development of programs across several intracellular target classes.
The biotech’s “delivery vehicles boast improved bioavailability, biodistribution, metabolic stability and enhanced efficiency compared to previous gold standards. The company is applying its platform to the delivery of intracellular enzyme replacement therapies, protein-protein interaction inhibitors and nucleic acids.”
Entrada is working on treating a rare and fatal mitochondrial disease caused by mutations in a gene that codes for an intracellular enzyme that has no approved therapy. With the Series A financing, the company hopes to advance this platform into human clinical trials.
According to the Xconomy website, Entrada has tested its approach in mice. The tests show that the company’s platform can get a therapy into a cell, reach multiple organs, and remain stable in the body.
In explaining Entrada’s approach, the Boston Globe said the company hopes to use peptides — short chains of amino acids — to deliver drugs produced from living organisms into cells.
Doshi described their approach as a potential game changer “for something that is so important across different disease states. That’s the beauty of the platform. It’s very flexible.”
By employing this approach, Entrada hopes it can treat more diseases. The company hopes that the Series A financing can be used to broaden its rare, monogenic disease pipeline by identifying and progressing additional intracellular enzyme replacement therapies.
Kush Parmar, the managing partner at 5AM Ventures said Entrada had an executive who was capable of “tackling the challenges posed by the delivery of biologics into the cell. Entrada is focused on leveraging a highly validated biological mechanism to fundamentally change the standard of care for several of these diseases via multiple intracellular targets.”
Todd Foley, managing director at MPM Capital, said: “Entrada’s unique vision to transform the treatment paradigm of certain diseases via intracellular biologics delivery could result in innovative treatments for thousands of patients. While there has been extensive development of biologics that address extracellular targets, there is also a significant opportunity for novel biologics that address intracellular targets. Entrada’s strategy and technology address this profound unmet need.”
The past few months have been particularly lucrative for startups working on therapies for rare diseases. In July, Healx, a four-year-old Cambridge (United Kingdom) technology company that develops innovative drugs for rare diseases, announced a $10 million Series A funding round, led by Balderton Capital.
Swiss-based biotech company, Therachon, which focuses on developing rare genetic disease treatments, closed a $60 million round of funding, with the company moving closer to a drug trial. Also, RDMD, a San Francisco based healthcare technology company that aims to advance research for patients with rare diseases, announced that it had raised $3 million in seed financing.