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The biotechnology and pharmaceutical industries are highly competitive. In particular, the field of gene therapy is characterized by
rapidly advancing technologies, intense competition and a strong emphasis on proprietary products. We face substantial competition from many different sources, such as large and specialty
pharmaceutical and biotechnology companies, academic research institutions, government agencies and public and private research institutions.
this time, there is no FDA- or EMA-approved treatment for SMA. However, a number of companies are developing drug candidates for SMA. We are aware of other companies exploring gene
therapy treatments in preclinical development, including Voyager Therapeutics, Inc. and Généthon. In addition to a gene therapy-based solution, alternative
approaches in clinical development for the treatment of SMA include alternative splicing and neuroprotection technologies. Potential competitors
- Alternative splicing seeks to achieve more efficient production of full-length SMN
protein from the SMN2 backup gene. Companies utilizing this approach include Ionis Pharmaceuticals, Inc., which, together with its licensee Biogen, is conducting Phase 3 clinical trials
across all SMA types, PTC Therapeutics and Roche Holding Ltd, which is conducting Phase 1 clinical trials across all SMA types and Novartis Corporation, which is conducting
Phase 2 clinical trials for SMA Type 1 patients in the EU.
- Neuroprotectants seek to mitigate the loss of motor neurons. Trophos SA, which
has been acquired by Roche Holding Ltd, has completed its Phase 3 clinical trial of its lead neuroprotectant product candidate, Olesoxime (TRO19622), in patients between the ages of
three and 25 with SMA Types 2 and 3.
We strive to protect and enhance the proprietary technology, inventions, and improvements that are commercially important to the
development of our business. We intend to seek, maintain and defend our patent rights, whether developed internally or licensed from third parties. We also rely on trade secrets relating to our
proprietary technology and on know-how, continuing technological innovation and in-licensing opportunities to develop, strengthen and maintain our proprietary position in the field of gene therapy.
Additionally, we intend to rely on regulatory protection afforded through orphan drug designations, data exclusivity and market exclusivity as well as patent term extensions, where available.
future commercial success depends, in part, on our ability to: obtain and maintain patent and other proprietary protection for commercially important technology, inventions and
know-how related to our business; defend and enforce licensed patent rights, and, if sought and obtained in the future, any patent rights we may own; preserve the confidentiality of our trade secrets;
and operate without infringing the valid enforceable patents and proprietary rights of third parties. Our ability to stop third parties from making, using, selling, offering to sell or importing our
current product candidate and any future product candidates may depend on the extent to which we have rights under valid and enforceable patents or trade secrets that cover these activities. With
respect to our licensed intellectual property, we cannot be sure that patents will issue with respect to any of the pending patent applications to which we license rights. With respect to any patent
applications that we or our licensors may file in the future, if any, we cannot be sure that any such applications will issue or, if issued, that such applications will be commercially useful in
protecting our product candidate and methods of manufacturing the same, or otherwise provide any competitive advantage. Even with respect to issued patents that we currently license, we cannot be sure
that any such patents will provide any competitive advantage. See "Risk factors Risks related to our