Viral Vectors Expected to Play Pivotal Role in Gene Therapy Pipeline for Neurology Indications


Viral vectors, such as adeno-associated virus (AAV), are expected to play a pivotal role in gene therapies for neurology indications, as this vector type has been found to be the most dominant among pipeline products classified under gene therapies, according to GlobalData, a leading data and analytics company. 

Vinie Varkey, Neurology and Ophthalmology Senior Analyst at GlobalData commented “Of the 38 pipeline products that are currently in development stages (Phase I, II, and III), AAV-based delivery platforms account for 45%. Lentiviral, the other viral-based platform, accounts for 13% of the total products.”

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Among non-viral-based products, antisense oligonucleotides (ASOs) dominate by representing 21% of the total products. The success of ASOs has largely been due to the mechanism of action (MOA), which has been demonstrated through the commercial success of Biogen’s Spinraza, the first-to-market drug for treatment of spinal muscular atrophy (SMA). Therapies with similar MOAs are currently being evaluated in other indications such as Alzheimer’s disease, amyotrophic lateral sclerosis, Huntington’s disease, migraine and multiple sclerosis. Figure 1 shows the percentage of pipeline products by type of delivery platform.

Varkey adds “AAV-based viral therapies recently witnessed a milestone when the FDA approved Novartis’ Zolgensma in May 2019 for the treatment of children with SMA. The therapy is proposed to work as a one-off therapy to introduce the missing gene that can help in production of the protein that is otherwise missing in patients with SMA.”

As clinical efficacy of AAV-based vectors becomes more established, there is an expectation that applications of these vectors will extend to other neurology indications. Viral vectors are understood to have higher transfection efficacy compared to non-viral vectors, whereas the latter is often associated with a better safety profile.

Varkey concludes: “As these pipeline therapies progress through different phases of clinical development, an increased understanding of the different MOAs is expected to lead to an increase in the use of these vectors for various indications.”

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