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Vertically integrated clinical stage gene therapy company: MeiraGTx Holdings plc (MGTX)

MeiraGTx Holdings plc (NASDAQ:MGTX) was incorporated in the Cayman Islands on May 1, 2018. It is a holding company to take over the assets of the British subsidiary MeiraGTx Limited. It is headquartered in London, England, and its main administrative office is in New York City, New York. With 59 full-time employees (3/31/2018), it is a vertically integrated, clinical-stage, gene therapy company with four ongoing clinical projects and a series of pre-clinical and research projects.

MeiraGTx

MeiraGTx Holdings plc (MGTX):

MeiraGTx Holdings plc, registered in the Cayman Islands, is the holding parent company of British gene therapy company MeiraGTx Limited. MeiraGTx has core capabilities in viral vector design and optimization, gene therapy manufacturing, and potential transgenic gene regulation technology. Under the leadership of an experienced management team, MeiraGTx has adopted a combined approach through licensing, acquisition and development of technologies, so that the company can have an in-depth understanding of candidate products and indications.

MeiraGTx initially focused on gene therapy research for ophthalmology, salivary glands and central nervous system diseases. Currently, the company plans to expand its research fields to develop gene therapy treatments for patients with a series of serious diseases.

MeiraGTx owns and operates a flexible and scalable viral vector manufacturing plant. The company expects to be approved by regulatory authorities to produce vectors for use in the company’s clinical and preclinical projects. If approved, it can provide sufficient commercial production capacity (capacity).

MeiraGTx completed the design in early 2018 to meet global regulatory requirements, including the current Good Manufacturing Practices (cGMP) required by the U.S. Food and Drug Administration (FDA). The company’s 29,000 square foot facility has 2 enclosed production suites (2 cell production suites), 3 independent viral vector production kits, providing multi-product and multi-viral vector manufacturing capabilities, and an integrated, flexible filling and completion kit. In May 2018, MeiraGTx obtained a license from the British Pharmaceutical and Healthcare Products Regulatory Agency to produce gene therapy candidates in the company’s cGMP-compliant production facilities.

MeiraGTx has also established a comprehensive platform to provide support for the efficient clinical development of next-generation gene therapies and production under cGMP requirements. MeiraGTx’s in-depth understanding of disease models provides a powerful analysis method for the cGMP production of its candidate products. The company’s team has rich experience in viral vector design and works closely with its process development team to design viral vectors and develop proprietary production cell lines , In order to effectively expand the manufacturing process.

MeiraGTx is also developing a potentially transformative technology that uses small molecules as switches for gene therapy products. The goal of this gene regulation platform is to transform gene therapy into a generalized delivery mechanism of biological drugs that uses small molecule “switches” for time control. MeiraGTx believes that the time control capabilities of gene therapy products may change the field of gene therapy by opening up new therapeutic possibilities.

MeiraGTx Holdings plc (MGTX) candidate products:

1. AAV-CNGB3 ——MeiraGTx has developed a candidate gene therapy product AAV-CNGB3 for the treatment of color blindness (Achromatopsia, ACHM) caused by mutations in the CNGB3 gene. The company is conducting Phase I/II clinical trials of AAV-CNGB3 in adult and pediatric patients.

2. AAV-CNGA3- MeiraGTx is developing a candidate gene therapy product AAV-CNGA3 to treat color blindness (ACHM) caused by mutations in the CNGA3 gene. The company plans to conduct Phase I/II clinical trials of AAV-CNGB3 in adult and pediatric patients.

3. AAV-RPE65- MeiraGTx has developed a candidate gene therapy product AAV-RPE65 for the treatment of RPE65 defects (RPE65 defects cause rod photoreceptive dysfunction and impaired vision, and there are approximately 125,000 patients in the United States). The codon-optimized RPE65 gene is driven by a novel synthetic retinal pigment epithelial cell-specific promoter. The company is conducting Phase I/II clinical trials of AAV-RPE65 in adult and pediatric patients.

4. AAV-RPGR ——MeiraGTx has developed a candidate gene therapy product AAV-RPGR for the treatment of XLRP (X-Linked Retinitis Pigmentosa), which aims to treat RPGR genes (Retinitis Pigmentosa). The most common form of XLRP caused by mutations in the eye-specific form of enzyme-regulated genes is called RPGR-ORF 15. The company is conducting Phase I/II clinical trials of AAV-RPGR in adult and pediatric XLRP patients with RPGR-ORF 15 mutations.

5. AAV-AQP1- MeiraGTx has developed a candidate gene therapy product, AAV-AQP1, for the treatment of grade 2 or grade 3 RIX (Radiation Induced Xerostomia, radiation-induced xerostomia). Currently, MeiraGTx is conducting a phase I/II clinical trial of AAV-AQP1 for use in patients with cancer-free survival 5 years or more after treatment for head and neck cancer, and patients with grade 2 or grade 3 RIX. .

6. Pre-clinical candidate products, including: AAV-UPF1, Wet AMD and Dry AMD, etc.

meiragtx pipeline

MeiraGTx Holdings plc (MGTX) investment:

MeiraGTx Holdings plc (NASDAQ:MGTX) submitted its IPO prospectus on 5/14/2018, and listed on NASDAQ on 6/8/2018 with an issue price of US$15.00, issuing 5 million shares, and raising US$75 million.

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