The FDA offered recommendations for sponsors that do trials involving antisense oligonucleotide (ASO) products developed for use against life-threatening genetic diseases in a draft guidance released last week.

The guidance covers ethical and human subject protection considerations, diagnostic and genetic considerations, dosing considerations, drug product administration procedures, safety assessments and assessing clinical response.

For example, sponsors should make certain considerations when characterizing the gene variants being targeted and the trial participant’s clinical diagnosis and provide all relevant information in the IND submission, the guidance advises.

These include confirming the subject’s clinical and genetic diagnosis through laboratory testing (gene sequencing, enzymatic analysis, biochemical testing and imaging evaluations as they apply) and providing evidence that establishes the role of the genetic variant(s) targeted by the ASO drug product in the way the disease develops (pathogenesis).

Read the clinical draft guidance here: https://bit.ly/3EJPjdf.