New biotech gene therapy Telaria launches with focus on rare skin diseases - MedCity News

A new biotech company has been set up to develop a potential gene therapy for a rare, inherited disease that results in skin so fragile it tears as easily as paper.

Telaria is the newest company revealed by Replay, which itself is a novel biotechnology for gene therapy. Launched earlier this year, Replay operates with a “hub and spoke” business model in which it develops its own platform technologies and identifies therapeutic areas that can be addressed with one or more of those technologies. Replay, which maintains operations in San Diego and London, then formed companies focused on specific therapeutic areas.

Rare skin diseases are the focus of Telaria. The company’s lead program is being developed for recessive dystrophic epidermolysis bullosa (RDEB), an inherited disease in which genetic mutations block or disrupt the formation of collagen that connects the epidermis to the dermis. In the absence of connections between these layers, the skin becomes easily susceptible to wounds and blisters. There are no FDA-approved therapies for the disorder. Treatment is mainly supportive care. Compared to another type of disease called dominant dystrophic epidermolysis bullosa, RDEB is more severe and can cause scarring, severe pain, and internal organ problems. Complications that can result from RDEB include chronic inflammation and squamous cell carcinoma, a type of skin cancer.

Like other Replay companies, Telaria will use the greater capacity of the herpes simplex virus (HSV) to deliver gene therapy. Adeno-associated viruses (AAVs), often used for gene drug delivery, have a limited capacity for their genetic cargo. Replay has developed a version of HSV that it claims is capable of delivering up to eight times the payload capacity of AAV vectors. This capacity allows Replay to view genes that are too large for AAV-based delivery. The company is aiming for even bigger genes. He is developing an HSV vector that can deliver up to 30 times more payload than AAV.

“There is currently no approved treatment for people suffering from RDEB, and I am aware, from my personal experience, that the current standard of care is limited and does not provide long-term and sustainable benefit to patients,” Alexander, co-founder of Telaria Silver said in a prepared statement . “Replay’s synHSV technology, which enables the delivery of large DNA to the skin through its differentiated payload capacity, has the potential to disrupt the field of gene therapy for genetic skin diseases and deliver much-needed treatments to patients as quickly and safely as possible.”

Telaria joins a small group of companies dealing with the treatment of epidermolysis bullosa. Krystal Biotech developed B-VEC, a topical and reusable gene therapy designed to deliver two copies of the COL7A1 gene, which encodes a protein key to collagen formation. The Pittsburgh-based company’s gene therapy is currently under FDA review; a regulatory decision is expected by the end of February. Abeona Therapeutics is developing an RDEB cell therapy called EB-101. Earlier this month, the New York-based biotech reported positive phase 3 data, paving the way for plans to submit a biologics license application to the FDA. Amicus Therapeutics entered the pursuit of epidermolysis bullosa in 2015 with the acquisition of Scioderm, a biotech company developing a topical wound healing drug. But Amicus ultimately halted development of that program two years later after failure of a phase 3 clinical trial.

Replay launched in July, backed by $55 million in seed funding led by KKR & Co. and OMX Ventures. Telaria is the second Replay company introduced this year, following last month’s launch of Retinal Eye Disease Eudora Biotechnology. This company is developing gene therapies for retinitis pigmentosa, Stargardt disease, and Usher syndrome type 1B.

Photo, public domain by CDC

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