This week's Philadelphia-area life sciences news includes a potential new pain medicine, a gene therapy candidate, a key patent secured by a gene therapy company, and more.
Here's the roundup:
Trevena
The Chesterbrook biopharmaceutical company is advancing TRV045, a potential treatment for diabetic neuropathic pain, into clinical development.
Trevena's announcement followed its receipt of a notification from the Food and Drug Administration informing the company that its planned three-part Phase 1 study may proceed.
“[Diabetic neuropathic pain] is a painful condition that affects over 5 million people in the United States," said Carrie Bourdow, CEO of Trevena, noting the currently available therapies for the condition have both efficacy and tolerability concerns. "TRV045 represents a potentially new approach to treating this burdensome condition.”
TRV045 is a novel, selective S1P1 receptor modulator. S1P receptors are located throughout the body, including the central nervous system, where they are believed to play a role in modulating neurotransmission and membrane "excitability" — the property that allows a cell to generate an electrical signal in response to a stimuli such as pain.
Trevena (NASDAQ: TRVN), through a collaboration with the National Institutes of Health, is also evaluating TRV045 as a potential treatment for epilepsy.
Interius BioTherapeutics
The gene therapy company spun out of the University of Pennsylvania two years ago was granted a patent titled "The Methods and Composition for Gene Delivery Using an Engineered Viral Particle" from the U.S. Patent and Trademark Office.
Interius is attempting to develop genetic therapies that are potentially more effective, safer and more accessible to address diseases driven by single-gene mutations as well as complex diseases, such as cancers.
"Our task is very ambitious, but our team is committed to the deep understanding of every component of the modular gene therapy platform we are building," said Dr. Phil Johnson, CEO of Interius.
Securing the patent, Johnson said, takes the company "one step closer to our ultimate goal of one day making novel transformational treatments available to patients."
Interius was co-founded by Dr. Saar Gill, a physician-scientist and cell therapy pioneer at the University of Pennsylvania, and Bruce Peacock, a biotech industry veteran. The company is initially focused on treating hematologic malignancies.
Ocugen
The FDA accepted the Malvern biopharmaceutical company's investigation new drug application for OCU400.
The acceptance will allow Ocugen (NASDAQ: OCGN) to begin human testing of its experimental gene therapy, which is being developed to treat the eye disease retinitis pigmentosa resulting from genetic mutations.
OCU400, during the past two years, was granted four orphan drug disease designations from the FDA for treating four different gene mutation-associated retinal degenerative diseases. The designations provide Ocugen with incentives that include tax credits for qualified clinical trials, exemptions from user fees, and the potential for seven years of market exclusivity if a product receives regular approval.
The Orphan Drug program was established by the FDA to encourage companies to develop treatments for rare conditions that afflict fewer than 200,000 people nationwide.
Details on Ocugen's clinical trial, the company said, will be available in the coming weeks on www.clinicaltrials.gov.
Quick hits
Philadelphia-based iEcure entered into an exclusive agreement with the University of Pennsylvania to develop next-generation lipid nanoparticles for liver gene editing applications. Financial terms of the deal were not disclosed. The technology was developed in the laboratory of Michael Mitchell, an assistant professor of innovation in the Department of Bioengineering at the University of Pennsylvania School of Engineering and Applied Science. … Radnor-based chemicals and materials company Avantor (NYSE: AVTR) opened a single-use logistics hub in Westminster, Massachusetts. The company said it opened the plant, which will serve as a center for raw material storage, quality control and distribution, to strengthen its global biopharma supply chain for customers that include pharmaceutical and biotechnology companies. … Two local life sciences companies — Harmony Biosciences (NASDAQ: HRMY) of Plymouth Meeting and Century Therapeutics (NASDAQ: IPSC) of Philadelphia — were added to the NASDAQ Biotechnology Index.
