News

February 12, 2020February 12, 2020

Cannabinol Identified as Active Component of Potential EB Treatment INM-755, InMed Announces

Cannabinol (CBN), a rare type of cannabinoid, is the active component of InMed Pharmaceuticals‘ INM-755 treatment candidate for epidermolysis bullosa (EB). CBN is also the active component of InMed’s INM-088, being ... Read more

February 10, 2020February 10, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

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The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 7, 2020February 7, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

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The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

February 5, 2020February 5, 2020

Krystal Opens Second Facility to Support Production and Potential Commercialization of its Gene Therapy B-VEC

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Krystal Biotech is opening its second gene therapy facility, called Astra, to support the production and potential commercialization of B-VEC, the company’s gene therapy for people with dystrophic epidermolysis ... Read more

January 29, 2020January 29, 2020

Aegle Raises $4M to Fund First Phase 1/2 Trial of Experimental Therapy AGLE-102 for DEB

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Aegle Therapeutics has raised $4 million to fund the first clinical trial evaluating AGLE-102, a therapy candidate for people with dystrophic epidermolysis bullosa (DEB) based on the company’s extracellular ... Read more

January 22, 2020January 22, 2020

Abeona Launches Phase 3 Trial Testing EB-101 Cell Therapy for RDEB

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Abeona Therapeutics has begun its Phase 3 VIITAL trial evaluating EB-101, a gene-corrected cell therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), the company recently announced. An ... Read more

January 15, 2020January 15, 2020

Castle Creek Acquires Fibrocell, Now Leading Development of FCX-007 Gene Therapy for RDEB

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Castle Creek Pharmaceutical recently acquired Fibrocell Science and is now leading the development of treatments for epidermolysis bullosa (EB), including the investigational gene therapy FCX-007 for the treatment of recessive ... Read more

January 8, 2020January 8, 2020

Research Consortium Receives $1.6M to Develop Stem Cell Therapies for Skin Diseases

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The Epidermolysis Bullosa iPS Cell Consortium has received $1.6 million in funding to develop stem cell-based therapies for people with inherited skin diseases such as epidermolysis bullosa (EB). A grant ... Read more

December 18, 2019December 18, 2019

Spray Therapy APR-TD011 Earns FDA Orphan Drug Designation to Treat EB

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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to APR-TD011, a spray therapy candidate for the treatment of epidermolysis bullosa (EB). This designation is intended to encourage ... Read more

December 11, 2019December 11, 2019

FDA Clears Phase 3 Trial Testing EB-101 Cell Therapy for Severe Blistering Skin Disorder

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After its recent clinical hold by regulators, the Phase 3 VIITAL trial has been given the green light to investigate EB-101, a gene-corrected cell therapy for people with recessive dystrophic ... Read more

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