International Journal of Genetic Medicine and Gene Therapy

This industry update features a round-up of pharmaceutical news in May 2019 based on press releases and websites. The month was characterized by the achievement of significant milestones in gene therapy. The biggest of these was the US FDA’s approval of Zolgensma®. This medicine sums up the promise and price of genetic medicine. On one hand the clinical results show Zolgensma can dramatically improve the prognosis for infants with spinal muscular atrophy after just one administration, while on the other, it has been priced at around US$2.1 million. With more such therapies likely to reach the market, the debate on Zolgensma goes beyond cost, to overall affordability, the true meaning of cost–effectiveness and how to reward companies for effective, innovative medicines.

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Gene Therapy and Genetic Vaccination: Two Hallmarks of Genetic Medicine

Animal Cell Technology ◽

10.1007/978-94-011-5404-8_5 ◽

1997 ◽

pp. 33-35

Author(s):

Dalemans Wilfried

Keyword(s):

Gene Therapy ◽

Genetic Medicine

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Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors

Toxicologic Pathology ◽

10.1177/01926233211041962 ◽

2021 ◽

pp. 019262332110419

Author(s):

Julie A. Hutt ◽

Basel T. Assaf ◽

Brad Bolon ◽

Joy Cavagnaro ◽

Elizabeth Galbreath ◽

...

Keyword(s):

Gene Therapy ◽

Research And Development ◽

Molecular Techniques ◽

Therapeutic Modality ◽

Adeno Associated Virus ◽

Genetic Medicine ◽

Case Basis ◽

Regulatory Landscape ◽

Support Research

Sequencing of the human genome and numerous advances in molecular techniques have launched the era of genetic medicine. Increasingly precise technologies for genetic modification, manufacturing, and administration of pharmaceutical-grade biologics have proved the viability of in vivo gene therapy (GTx) as a therapeutic modality as shown in several thousand clinical trials and recent approval of several GTx products for treating rare diseases and cancers. In recognition of the rapidly advancing knowledge in this field, the regulatory landscape has evolved considerably to maintain appropriate monitoring of safety concerns associated with this modality. Nonetheless, GTx safety assessment remains complex and is designed on a case-by-case basis that is determined by the disease indication and product attributes. This article describes our current understanding of fundamental biological principles and possible procedures (emphasizing those related to toxicology and toxicologic pathology) needed to support research and development of in vivo GTx products. This article is not intended to provide comprehensive guidance on all GTx modalities but instead provides an overview relevant to in vivo GTx generally by utilizing recombinant adeno-associated virus-based GTx—the most common in vivo GTx platform—to exemplify the main points to be considered in nonclinical research and development of GTx products.

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