Junctional Epidermolysis Bullosa 1 is under clinical development by Holostem Terapie Avanzate and currently in Phase II for Epidermolysis Bullosa. According to GlobalData, Phase II drugs for Epidermolysis Bullosa have a 60% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Junctional Epidermolysis Bullosa 1’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Junctional Epidermolysis Bullosa 1 overview

Gene therapy is under development for the treatment of junctional epidermolysis bullosa (JEB). It consists of autologous cultured epidermal grafts containing epidermal stem cells genetically modified with the aid of a retroviral vector carrying LAMB-3 cDNA. It is administered through cutaneous route.

Holostem Terapie Avanzate overview

Holostem Srl (Holostem), a subsidiary of Enea Tech and Biomedical Foundation, is a clinical research and biotechnological company. The company focuses on development, manufacture, registration and distribution of advanced therapies medicinal products (ATMPs) based on cultures of epithelial stem cells both for cell and gene therapy. Its product portfolio includes epithelial stem cells, cell therapy, gene therapy, and regenerative medical products. The company service offerings include preclinical research, clinical trials, research and development, drug development, and drug discovery programs. It has an operational presence in Czech Republic. Holostem is headquartered in Modena, Emilia-Romagna, Italy.

For a complete picture of Junctional Epidermolysis Bullosa 1’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 10 June 2024

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GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.