User:Biologist122/sandbox

Sources for use in "Specific projects and Products" section of "Virotherapy" page:

• Imlygic (talimogene laherparepvec) intralesional injection, FDA Package Insert: https://www.fda.gov/media/94129/download
  • Approved 2015
  • FDA Medication Guide for additional info https://www.fda.gov/media/94135/download
• Zolgensma (onasemnogene abeparvovec-xioi) intavenous infusion, FDA Package Insert: https://www.fda.gov/media/126109/download
  • Approved 2019; https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease
  • Data problems in clinical trials; https://www.reuters.com/article/us-usa-fda-novartis/novartis-says-knew-about-zolgensma-data-problems-before-u-s-approval-idUSKCN1UX1KA
• LUXTURNA (voretigene neparvovec-rzyl) intraoocular injection, FDA Package Insert: https://www.fda.gov/media/109906/download
  • Approved 2017; https://www.fda.gov/news-events/press-announcements/fda-approves-novel-gene-therapy-treat-patients-rare-form-inherited-vision-loss

• Glybera
  • https://www.ema.europa.eu/en/documents/overview/glybera-epar-summary-public_en.pdf

History -- 2,335 clinical trials between 1989 & 2015 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5328344/

Plans for page improvement

• Main Focus: Update "Viral gene therapy" subsection of "Specific projects and products" section.
  • Previous content was outdated and lacked notability given the current state of the field
  • New content provides information related to viral gene therapy products that have been approved in the US and/or EU
• Ensure that information listed is backed by reputable sources with proper citations, with a focus on the "Viral gene therapy" section (currently has no citations)

• Minor rewording throughout to maintain encyclopedic writing style
• Addition of links to other Wikipedia articles where applicable
• Addition of images, specifically of viral gene therapy

Viral gene therapy

Viral gene therapy uses genetically engineered viral vectors to deliver a gene of interest to target cells. An AAV vector is shown in this example.

Viral gene therapy uses genetically engineered viral vectors to deliver therapeutic genes to cells with genetic malfunctions. Typically, the virus is administered to patients intravenously or by direct injection into target tissues. The molecular mechanisms of gene delivery and/or integration into cells vary based on the viral vector that is used.

The genetic targets of viral gene therapy are varied. While some therapies target the missing or mutated genes of inherited genetic disorders, others seek to deliver new genes into cancer cells in order to destroy tumors.

Currently, Immune responses to viral therapies pose a challenge to successful viral gene therapy treatment. However, immune response to viral vectors at immune privileged sites such as the eye may be reduced compared to other sites of the body.

Specific projects and products

Viral gene therapy

Currently, there are many viral gene therapy products in clinical trials phases. Listed below are the products that are (or were) approved for use in the US and or/ European Union.

• In 2012 the European Commission approved Glybera, an AAV vector-based gene therapy product for the treatment of lipoprotein lipase deficiency in adults . It was the first gene therapy approved in the EU . The drug never received FDA approval in the US, and was discontinued by its manufacturer uniQure in 2017 due to profitability concerns . It is no longer authorized for use in the EU.
• In 2017, the FDA approved Spark Therapeutics' Luxturna, an AAV vector-based gene therapy product for the treatment of RPE65 mutation-associated retinal dystrophy in adults. Luxturna is the first gene therapy approved in the US for the treatment of a monogenetic disorder. It has been authorized for use in the EU since 2018.
• In 2019, the FDA approved Zolgensma, an AAV vector-based gene therapy product for the treatment of spinal muscular atrophy in children under the age of two . As of August 2019, it is the world's most expense treatment, at a cost of over two million USD . Novartis is still seeking marketing approval for the drug in the EU as of 2019.

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