How Sanofi is dominating the Global Fabry Disease Treatment Market

The purpose of the strategic research study titled "Fabry Disease Treatment Market - GLOBAL Industry Perspective Comprehensive Analysis and Forecast 2020 - 2028" is to offer industry investors, company executives, and industry participants with in-depth analysis to enable them make informed strategic decisions related to the opportunities in the industry.

4D Molecular's lead product candidate for treatment of Fabry and other lysosomal storage disorders is 4D-310, which uses a proprietary vector design for efficient delivery to relevant tissues with minimal toxicity and resistance to pre-existing antibodies. The Company has completed a pre-IND discussion with the FDA and will initiate a phase 1/2 clinical trial for the H2,2020. The discussion was on the basis of an IND-enabling pharmacology and Good Laboratory Practices (GLP) toxicology and biodistribution study that was initiated in 2019.

The Company uses its proprietary mRNA platform to discover and create novel solutions across indications such as prophylactic vaccine development, personalized cancer vaccine, intra-tumoral immune-oncology and localized regenerative medicine to name a few. Moderna has developed a unique mRNA sequence that codes for a protein, and encompasses knowledge of mRNA biology, chemistry, formulation & delivery, bioinformatics and protein engineering to address unmet needs in critical areas.
The Platform offers great flexibility and wide applicability; by changing the sequence of the genetic code it can combine different mRNA sequences for different proteins in a single mRNA medicine. It has a drug design studio, which can develop mRNA within a very short period of time, using a proprietary Sequence Designer module, bioinformatics algorithms and its modular synthesis robotics, making it a pioneering Digital Biotech Company.

The Company is developing a treatment for Fabry disease as part of its systemic secreted therapeutics modality, besides developing therapeutics for methylmalonic acidemia (MMA), propionic acidemia (PA), glycogen storage disorder Type 1a (GSD1a) and through its systemic intracellular therapeutics modality. mRNA-3630 is currently undergoing pre-clinical evaluation and the Company intends to file for an IND soon.

Scientists and researchers are utilizing big efforts while searching for new treatment options for Fabry disease treatment. As per estimation, nearly billions of dollars are invested in developing a treatment through gene therapy. Unfortunately, a lot of that effectively goes down the drain as money spent on the nine out of ten therapies that fail somewhere between phase I trials and regulatory approvals. Gene therapy for Fabry disease is designed to introduce a healthy copy of the GLA gene inside cells to restore normal levels of the alpha-galactosidase-A enzyme. This approach is still in the experimental stages.
Gene Therapy for Fabry Disease-
• FLT190- It is an experimental gene therapy which is being developed by Freeline. This study is currently in Phase 1/2 clinical trial (NCT04040049) and is currently recruiting up to 15 patients of age 18 and older, at sites in the U.K., Norway, and Italy.
• ST-920- This experimental gene therapy is being developed by Sangamo Therapeutics. Currently in Phase 1/2 clinical trial (NCT04046224), this study is recruiting about 30 adults with across 6 sites in the U.S. to test the safety and efficacy of ascending doses of a single infusion of this therapy.

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