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Copied from: https://www.folliclethought.com/crispr-cas9-therapy-for-androgenic-alopecia-moogene-medi/
CRISPR/Cas9 Therapy For Androgenic Alopecia: Moogene Medi
Moogene Medi of South Korea is the first company to formally introduce a CRISPR-based gene editing hair loss therapy to the world.
Description Of CRISPR Therapy For AGA
Moogene has a developed a microbubble-nanoliposomal delivery system to get gene editing particles directly into the dermal papilla cells of the hair follicle. First, a sequence of sgRNA or “guide RNA” is created to target the SRD5A2 gene; this is the gene responsible for creating the 5AR enzyme which converts testosterone into DHT, the known core driver of androgenic alopecia. The combination of Cas9/sgRNA, which is designed to suppress the SRD5A2 gene, is then encapsulated into nanoliposomes which are attached to microbubbles (see image below for depiction) within a topical solution. The topical solution is then applied to skin containing hair follicles (it has so far been tested on mice, and next will be tested on non-human primates). Then, ultrasound of a specific frequency is applied to the skin which bursts the microbubbles and creates momentary cavities in the surface of the skin to transport the nanoliposomes through the epidermis and into the dermal papilla bulb/cells. The Cas9 protein acts as the scissors to cut into the native RNA and allows the crafted sgRNA sequence to suppress the 5AR gene within the dermal papilla. The hair follicle cells, thus, are modified to be theoretically genetically exempt from DHT interference.
Moogene Microbubble Containing Nanoliposomes Around Outer Edge
Clinical Development Timeline
Currently, Moogene’s therapy for androgenic alopecia is the second highest priority at the company and only behind a treatment for colon cancer. Also, both of those programs will be worked on simultaneously which is good news. The next development step for Moogene’s AGA therapy is to complete a toxicity study for the FDA. This study will likely be done on a non-human primate species and is a measure taken to ensure that the Cas9/sgRNA particles do not migrate and produce toxic results in other areas of the body. To prove this in a toxicity study, tissue assays are taken from various organs and analyzed for traces of the Cas9/sgRNA therapy. I am told by Moogene co-founder Emmanuel Hui that if successful in the toxicity study, this treatment could move into human trials in about 2 years time. I also received from Emmanuel a PDF to share with the audience which contains a summary of Moogene’s peer-reviewed study which appeared in the February 2020 edition of the Biomaterials journal.
Partnerships For The Ultrasound-Microbubble Technology
Hui also tells me that his company is interested in partnerships with larger pharmaceutical companies that could accelerate the development of their AGA gene editing therapy. In addition to the gene editing therapy, Moogene also has filed patents for the delivery of more common drugs to treat hair loss via their ultrasound-microbubble technology. For example, molecules like finasteride or dutasteride could be delivered more effectively into the dermal papilla cell region of the hair follicle with this technology. At this time, Moogene is very happy to connect with any companies who would be interested in developing their technology with approved hair loss drugs.
Discussion
Since the inception of gene editing therapies, the idea of a gene therapy for hair loss has been tossed around. People theorized it could be an ideal way to create a mostly “full-proof” solution to hair loss. Although there is certainly much important testing to be done and years ahead, the fact that an established therapy candidate like Moogene’s sits in a pipeline is an accomplishment. In my discussion with Emmanuel Hui, co-founder of this technology, he was keen to point out that suppressing a gene responsible for DHT within the DP cells has potential to be much more efficacious than DHT blockers which work systemically. Thus, he also thought the potential for regrowth in dormant scalp areas from gene therapy is much higher than with finasteride, for example, which has shown the ability to regrow some lost hair for many of its users. Finally, to the question of how long this treatment will last or how many times it needs to be applied if it does work, Hui did not want to go on record with a specific prediction, he does describe the treatment as long-lasting and based on the impression I got, this audience would be pleased about the potential frequency of treatments needed.
Thanks to Emmanuel Hui for taking the time to share with us and Best Wishes to the Moogene company as they develop this first-of-its-kind gene therapy for hair growth.
CRISPR/Cas9 Therapy For Androgenic Alopecia: Moogene Medi
Moogene Medi of South Korea is the first company to formally introduce a CRISPR-based gene editing hair loss therapy to the world.
Description Of CRISPR Therapy For AGA
Moogene has a developed a microbubble-nanoliposomal delivery system to get gene editing particles directly into the dermal papilla cells of the hair follicle. First, a sequence of sgRNA or “guide RNA” is created to target the SRD5A2 gene; this is the gene responsible for creating the 5AR enzyme which converts testosterone into DHT, the known core driver of androgenic alopecia. The combination of Cas9/sgRNA, which is designed to suppress the SRD5A2 gene, is then encapsulated into nanoliposomes which are attached to microbubbles (see image below for depiction) within a topical solution. The topical solution is then applied to skin containing hair follicles (it has so far been tested on mice, and next will be tested on non-human primates). Then, ultrasound of a specific frequency is applied to the skin which bursts the microbubbles and creates momentary cavities in the surface of the skin to transport the nanoliposomes through the epidermis and into the dermal papilla bulb/cells. The Cas9 protein acts as the scissors to cut into the native RNA and allows the crafted sgRNA sequence to suppress the 5AR gene within the dermal papilla. The hair follicle cells, thus, are modified to be theoretically genetically exempt from DHT interference.
Moogene Microbubble Containing Nanoliposomes Around Outer EdgeClinical Development Timeline
Currently, Moogene’s therapy for androgenic alopecia is the second highest priority at the company and only behind a treatment for colon cancer. Also, both of those programs will be worked on simultaneously which is good news. The next development step for Moogene’s AGA therapy is to complete a toxicity study for the FDA. This study will likely be done on a non-human primate species and is a measure taken to ensure that the Cas9/sgRNA particles do not migrate and produce toxic results in other areas of the body. To prove this in a toxicity study, tissue assays are taken from various organs and analyzed for traces of the Cas9/sgRNA therapy. I am told by Moogene co-founder Emmanuel Hui that if successful in the toxicity study, this treatment could move into human trials in about 2 years time. I also received from Emmanuel a PDF to share with the audience which contains a summary of Moogene’s peer-reviewed study which appeared in the February 2020 edition of the Biomaterials journal.
Partnerships For The Ultrasound-Microbubble Technology
Hui also tells me that his company is interested in partnerships with larger pharmaceutical companies that could accelerate the development of their AGA gene editing therapy. In addition to the gene editing therapy, Moogene also has filed patents for the delivery of more common drugs to treat hair loss via their ultrasound-microbubble technology. For example, molecules like finasteride or dutasteride could be delivered more effectively into the dermal papilla cell region of the hair follicle with this technology. At this time, Moogene is very happy to connect with any companies who would be interested in developing their technology with approved hair loss drugs.
Discussion
Since the inception of gene editing therapies, the idea of a gene therapy for hair loss has been tossed around. People theorized it could be an ideal way to create a mostly “full-proof” solution to hair loss. Although there is certainly much important testing to be done and years ahead, the fact that an established therapy candidate like Moogene’s sits in a pipeline is an accomplishment. In my discussion with Emmanuel Hui, co-founder of this technology, he was keen to point out that suppressing a gene responsible for DHT within the DP cells has potential to be much more efficacious than DHT blockers which work systemically. Thus, he also thought the potential for regrowth in dormant scalp areas from gene therapy is much higher than with finasteride, for example, which has shown the ability to regrow some lost hair for many of its users. Finally, to the question of how long this treatment will last or how many times it needs to be applied if it does work, Hui did not want to go on record with a specific prediction, he does describe the treatment as long-lasting and based on the impression I got, this audience would be pleased about the potential frequency of treatments needed.
Thanks to Emmanuel Hui for taking the time to share with us and Best Wishes to the Moogene company as they develop this first-of-its-kind gene therapy for hair growth.
