Please tell us about your clinical experience. How many patients have you had go through gene therapy? What has the experience been like, from their perspective, and yours?

Can you walk us through the following gene therapy techniques:-Gene augmentation therapy-Gene inhibition therapy-Killing of specific cellsWhat’s the use case where each of those would make sense?

In 2020, 6 cell and gene therapy programs were forced to delay their development timelines after the FDA asked for more information about their production processes. How important is this aspect in your opinion?

Could you discuss any of the following gene therapy variations: tumour suppressor, suicide, antiangiogenesis, inflammatory cytokine and micro-RNA genes, new vectors such as oncolytic viruses, and the synergy between viral gene therapy, chemotherapy and radiation therapy?

 The FDA predicts that by 2025 10 to 20 cell and gene therapies will get approved every year. Considering that about 5% of the roughly 7,000 rare diseases have an FDA-approved drug, how well do you think the companies active in this space can adapt to those unmet needs?

In 2020, 6 cell and gene therapy programs were forced to delay their development timelines after the FDA asked for more information about their production processes. Why is this such a prevailing issue, in your opinion and how much can it stall growth in the industry?

Could you discuss any of the following gene therapy variations: tumour suppressor, suicide, antiangiogenesis, inflammatory cytokine and micro-RNA genes, new vectors such as oncolytic viruses, and the synergy between viral gene therapy, chemotherapy and radiation therapy?

The FDA predicts that by 2025 10 to 20 cell and gene therapies will get approved every year. Considering that about 5% of the roughly 7,000 rare diseases have an FDA-approved drug, how well do you think the companies active in this space can adapt to those unmet needs?

How often is it the case that gene therapy can only alleviate symptoms and not eliminate the underlying cause? How often is a complete cure achievable with gene therapies?

How do you deal with side effects that can result from cytokine release syndrome, following gene therapy administration? How well has management of side effects improved in recent years?

What existing gene therapies work really well? What upcoming treatments do you have high expectations for?

Adverum Biotechnologies (ADVM) stopped testing one of its gene therapies this summer for a diabetic eye disease, after encountering safety problems. What chances of succes do you think REGENXBIO's Subretinal RGX-314 Gene Therapy Phase 1/2a Study for the Treatment of nAMD still has, in this space?
How common/ problematic are safety and AE issues for eye gene therapies?