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Discovery & Development Advanced Medicine, Drug Discovery, Clinical Trials

Perusing the Pipeline

Preclinical

Scientists have developed a gene therapy that prevents axon destruction in mice. When an axon is damaged, either through injury or by certain therapeutic drugs, a protein called SARM1 becomes active, which triggers axons to self-destruct. This destruction likely plays an important role in multiple neurodegenerative conditions, including peripheral neuropathy, Parkinson's disease and amyotrophic lateral sclerosis. The researchers used an AAV vector to introduce point mutations into human SARM1 and inhibit its function. They found axon preservation similar to that observed in SARM1 knockout mice (1).

An international team of researchers have used gene therapy to restore hearing in an adult mouse model of DFNB9 deafness – a hearing disorder that represents one of the most frequent cases of congenital genetic deafness in humans. Individuals with DFNB9 deafness are deficient in the gene coding for otoferlin, a protein essential for transmitting sound information at auditory sensory cell synapses. The researchers used a single intracochlear injection of two different recombinant AAV vectors to reconstruct the otoferlin coding region, leading to long-term restoration of otoferlin expression in the inner hair cells, and restored hearing (2).

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  1. Washington University School of Medicine in St. Louis, “Gene therapy blocks peripheral nerve damage in mice” (2019). Available at: https://bit.ly/2SHkCME.
  2. EurekAlert, “Gene therapy durably reverses congenital deafness in mice” (2019). Available at: https://bit.ly/2Tts96d.
  3. Sickle Cell Anemia News, “CTX001” (2019). Available at: https://bit.ly/2XDNlFV.
  4. ClinicalTrials.gov, “A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease” (2019). Available at: https://bit.ly/2GXidfI.
  5. Pharmaphorum, “Cell therapy produces encouraging first results in eye trial” (2019). Available at: https://bit.ly/2tBklB5.
  6. ClinicalTrials.gov, “Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)” (2019). Available at: https://bit.ly/2IOe4MI.
  7. Muscular Dystrophy News Today, “Gene Therapy SGT-001 Shows Signs of Microdystrophin Production in Muscles of Boys in IGNITE DMD Trial” (2019). Available at: https://bit.ly/2Tx3xtm.
  8. ClinicalTrials.gov, “CD123/CLL1 CAR-T Cells for R/R AML” (2019). Available at: https://bit.ly/2EKN78G.
  9. ClinicalTrials.gov, “HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients” (2019). Available at: https://bit.ly/2NFUHEl.
  10. Hemophilia News Today, “Single Dose of Gene Therapy AMT-061 Proves Effective in Hemophilia B” (2019). Available at: https://bit.ly/2XCnG0s.
  11. ClinicalTrials.gov, “A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)” (2019). Available at: https://bit.ly/2tRCgne.
  12. ClinicalTrials.gov, “Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product” (2019). Available at: https://bit.ly/2VAmGIr.

About the Author

James Strachan

Over the course of my Biomedical Sciences degree it dawned on me that my goal of becoming a scientist didn’t quite mesh with my lack of affinity for lab work. Thinking on my decision to pursue biology rather than English at age 15 – despite an aptitude for the latter – I realized that science writing was a way to combine what I loved with what I was good at.

From there I set out to gather as much freelancing experience as I could, spending 2 years developing scientific content for International Innovation, before completing an MSc in Science Communication. After gaining invaluable experience in supporting the communications efforts of CERN and IN-PART, I joined Texere – where I am focused on producing consistently engaging, cutting-edge and innovative content for our specialist audiences around the world.

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