Expert Interview
Angelman Syndrome Pipeline: Recent Updates on GTX-102 (Ultragenyx & GeneTx), ION582 (Ionis Pharmaceuticals), and rugonersen.
Ticker(s): OAK HILL BIO, IONS, RAREInstitution: University of California San Diego
- Dysmorphologist and clinical geneticist at Rady Children's, and a professor of clinical pediatrics at UC San Diego.
- Medical director of Rady Children's multidisciplinary Prader-Willi Syndrome Clinic and co-founder and medical administrator of Rady Children's Down Syndrome Center.
- Research focuses on Angelman syndrome, and she is the principal investigator in San Diego of a multi-site consortium that conducts a natural history study and clinical trials.
Roughly how many patients with Angelman syndrome do you currently manage?
Added By: sara_adminWhat are your thoughts on the recent data presented on ION582?
Added By: sara_adminWhat are your thoughts on the recent data presented on GTX-102?
Added By: sara_adminWhat are the main challenges in diagnosing Angelman syndrome, and how does insurance coverage impact this?
What are your thoughts on the safety and efficacy of ION582 compared to other ASOs for Angelman syndrome, and what might explain the discontinuation of Roche's rugonersen despite promising data?
Is there room for more than one approved ASO for AS?
Added By: user3ea366b7All the companies have generated EEG biomarker data. Any thoughts on what this shows?
Added By: user3ea366b7What are your thoughts on the data that Roche presented at ASF and FAST?
Added By: user3ea366b7Anecdotally, how would you describe the responses you've seen to the different drugs?
Added By: user3ea366b7Have you seen any work done nonclinically for how the ASOs compare?
Added By: user3ea366b7how confident are you in ultragenyx's ability to manage safety? they saw toxicity at lower doses than ionis and roche.
Added By: user3ea366b7were there differences in how the tests / assessments were performed for the different trials? did all use the same standards for vineland? bayley?
Added By: user3ea366b7how would you evaluate the trade-off in safety and efficacy? would you generally want to push the dose to get to greater efficacy, even if it increases some of the side effects seen, like vomiting and pyrexia?
Added By: user3ea366b7what are your plans for participation in the phase 3 trials?
Added By: user3ea366b7assuming an ASO is approved, any feeling for what percent of your patients would be interested in treatment?
Added By: user3ea366b7Are You Interested In These Questions?
Slingshot Insights Explained
Expert research benefits investors by giving them timely access to unbiased real world perspectives on highly specialized topics. Slingshot Insights' crowdfunded model makes this access available at a fraction of the cost of other expert networks.
Reason
*Slingshot Insights provides access to information, not investment advice. We work to support you and facilitate access to experts; however we are not responsible for monitoring calls for the disclosure of MNPI. You should obtain financial, legal and tax advice from your qualified and licensed advisers before deciding to invest in any security.
