Facilitating Gene Therapy Advancements

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On July 21, 2023, the Government of Canada registered Order 2023-112-08-01, amending the Domestic Substances List (DSL) under the Canadian Environmental Protection Act, 1999 (CEPA). This amendment introduces a specific living organism: the recombinant and non-replicating adeno-associated virus serotype 5 (AAV5) vector, which expresses the retinitis pigmentosa GTPase regulator gene RPGR (AAV5-hRKp.RPGR). This decision came after a thorough assessment by the Minister of the Environment and the Minister of Health, who confirmed that the organism had either been manufactured or imported into Canada and met the required criteria for inclusion in the DSL.

The DSL serves as an inventory of substances already available in the Canadian marketplace, first established in 1994. Regular updates to the list ensure that new chemicals, polymers, and living organisms undergo assessments to identify potential risks to human health and the environment. The process for adding substances to the DSL is governed by various sections of CEPA, which mandate that new substances not already listed must undergo notifications and assessments.

In this case, the Ministers assessed data on 11 new substances—10 chemicals and polymers, along with the AAV5-hRKp.RPGR living organism. After the assessment period ended and no regulatory conditions were placed on the organism, it was deemed eligible for inclusion in the DSL under subsection 112(1) of CEPA. This change facilitates the use of the organism in various applications without imposing additional regulatory barriers, thereby promoting innovation and business activities in the field of biotechnology.

The main objective of this amendment is to incorporate the AAV5-hRKp.RPGR vector into the DSL, making it more accessible for use in research and commercial activities. By adding this substance, the government aims to foster ongoing advancements in biotechnology, particularly those that can have significant impacts on health. The inclusion of this vector is also expected to improve efficiency for companies that manufacture or import these substances, as they will no longer need to comply with the more demanding new substance notification regulations.

The recombinant and non-replicating adeno-associated virus serotype 5 (AAV5) vector expressing the retinitis pigmentosa GTPase regulator gene RPGR (AAV5-hRKp.RPGR) is a specialized viral tool used primarily in gene therapy. AAV5 is a type of virus that can deliver genetic material into cells without causing disease, making it a useful vehicle for therapeutic applications. Being non-replicating means that the vector does not multiply in the host cells, reducing the risk of unintended effects and making it safer for therapeutic use. Overall, this AAV5-hRKp.RPGR vector represents a promising approach in the field of gene therapy for treating specific inherited retinal diseases.

Canada (SOR/2023-166) August 2, 2023