New company launches to advance potential treatment for DEB

A new biotechnology company called Rinascera Therapeutics has launched to develop therapies for people with epidermolysis bullosa and other rare genetic skin diseases.

The company is working to advance two treatment candidates. RIN-002 is being developed for dystrophic epidermolysis bullosa (DEB), while RIN-001 is being developed for another genetic skin disorder called Gorlin syndrome.

“People living with diseases like Gorlin Syndrome and DEB are not asking for marginal improvements. They are asking for lives with less pain, fewer surgeries, fewer wounds, fewer limitations and more possibility,” Nichola Eliovits, co-founder and CEO of Rinascera Therapeutics, said in a press release announcing the company’s launch. “We created Rinascera to develop therapies that address the biological foundations of rare genetic diseases and give patients and their families a genuine second chance at life.”

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DEB is caused by mutations in the COL7A1 gene that result in too little collagen VII or collagen VII that does not work properly. This protein normally helps anchor the outer layer of skin to the layer beneath it and maintain the integrity of other tissues. With too little functional collagen VII, people with DEB are prone to blistering, scarring, and wounds that heal slowly or incompletely. Some people with DEB, especially those with more severe forms of the disease, may also experience additional symptoms that can include digestive complications, eye and bone problems, and a heightened risk of a type of skin cancer called squamous cell carcinoma.

RIN-002 is designed to provide the body with a functional version of collagen VII, addressing the underlying protein deficiency in DEB. The treatment is administered by infusion into the bloodstream, with the goal of delivering the therapeutic protein to affected skin and other tissues.

“With RIN-002, we seek to move DEB treatment beyond the individual wound by systemically replacing collagen VII across affected tissues,” Eliovits said.

RIN-002 was previously known as PTR-01. Rinascera acquired the therapy from Bridgebio as part of the company’s launch. Bridgebio previously tested the therapy in a Phase 1/2 trial (NCT03752905) involving 12 people with recessive DEB and a Phase 2 study (NCT04599881) involving six participants. Results from the early studies suggested the protein replacement therapy was generally safe and well tolerated. Findings from the small, open-label Phase 2 study also indicated that the treatment helped to ease pain and promote wound healing.

Initial funding for Rinascera’s launch was led by the venture capital firm Double Point Ventures.

“At Double Point Ventures, we invest in companies that we believe can fundamentally change the standard of care for patients,” said Dan Yadegar, managing partner at Double Point Ventures and a member of Rinascera’s board of directors. “Rinascera combines exceptional leadership, differentiated clinical-stage programs and a disciplined strategy focused on diseases with substantial unmet need and clear underlying biology. We believe RIN-001 and RIN-002 provide the foundation to build a durable rare disease company capable of delivering meaningful benefits for patients while creating significant long-term strategic value.”