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Krystal Biotech in Three Words: Delivering Gene Therapies
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Background
Drug delivery is about making sure the right therapeutic is in the right place at the right time. Given the complexities of biology, it’s actually not as straightforward as it sounds. Krystal Biotech is an especially clever company that has developed a proprietary drug delivery mechanism for gene therapies.
The company has successfully navigated the challenges of gene therapy delivery by developing a proprietary vector from HSV-1. This is a virus that is highly prevalent in humans, so our bodies tolerate it such that it can be used repeatedly for redosing. The viral nucleic acid machinery that delivers the gene therapy to the cell, does just that, without incorporating its own information. Finally, this HSV-1 vector can carry gene therapies that exceed 30KB. Given that the mean size of a gene is 24KB, this vector is able to support the greatest number of possibilities.
The company is using its vector to deliver gene therapies to treat rare diseases, in particular Dystrophic Epidermolysis Bullosa (DEB). People with this disease have mutations in the gene COL7A1 which forms an important skin protein. With the mutation, the skin is not anchored and patients develop blisters and sores as their skin tears easily like a butterfly’s wing. The company received FDA approval of Vyjuvek in May of this year. It recently announced that its Marketing Authorization Application (MAA) to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for Vyjuvek has been validated and is now under CHMP review for approval in Europe. It expects to receive an opinion in the second half of 2024.
The company has no plans to be a one-hit wonder. It is using its vector to develop therapies for cystic fibrosis, alpha-1 antitrypsin deficiency (AATD), and oncology and has started on the path of clinical trials and commercialization. The company also applies its gene therapy delivery technology to serve the large aesthetics market, effectively serving patients who want its products and patients who need them.
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Krystal Biotech in Three Words: Delivering Gene Therapies