Angelman Syndrome Market Assessment: Epidemiology, Treatment Landscape, Unmet Needs, Emerging Therapies, and Value & Access

Table of Contents

• A rare, neurogenetic disorder characterized by developmental delays, seizures, and a happy and excitable demeanor
• Table 1.1. AS molecular subtypes
• The role of UBE3A in the disease mechanism of AS
• Figure 1.1. The role of UBE3A in the hypothesized pathogenesis of AS

• Disease definition
• Figure 2.1. G6 diagnosed prevalent cases of Angelman Syndrome by region
• Table 2.1. Diagnosed incident and prevalent populations of AS in the U.S. and EU5
• AS epidemiology and subtypes
• Figure 2.2. Proportion of AS patients with each molecular subtype

• Diagnosis overview
• Figure 3.1. Diagnostic pathway for AS patients
• AS requires lifelong medication courses and therapy
• Table 3.1. Treatment goals for AS - physician versus caregiver priorities
• Figure 3.2. Proportion of AS patients with difficult-to-treat seizures
• AS treatment involves a multidisciplinary care team collaborating with caregivers
• Figure 3.3. AS care team roles and responsibilities
• AS key characteristics throughout life course
• Physician insights on current AS treatment approaches
• Key treatment dynamics that will shape disease management and novel therapy use in AS
• Table 3.2. Must-know AS market dynamics
• Large opportunity for a therapy that will address neurodevelopmental outcomes
• Figure 3.4. Important dynamics of AS market evolution

• Overview
• Table 4.1. Top unmet needs in AS
• Physician perspectives on unmet needs in AS

• Overview
• Table 5.1. Comparison of ongoing AS disease-modifying therapy clinical trials
• Pipeline overview continued
• Table 5.2. Comparison of ongoing AS non-disease-modifying therapy clinical trials
• Preclinical AS gene therapy and delivery platforms
• Figure 5.1. Novel “HItap” AAV delivery platform
• Table 5.3. Preclinical GTP-220 summary
• Preclinical AS gene therapy pipeline
• Table 5.4. Preclinical research on AS gene therapies
• Physician insights on gene therapy options
• Failed AS trials and important drivers of future AS product differentiation
• Figure 5.2. Ovid Therapeutics OV101 Phase 3 efficacy data
• Important drivers of future AS product differentiation
• Figure 5.3. Attributes that will drive uptake for emerging AS therapies

• Overview
• Table 6.1. Comparison of gene therapy pricing
• Overview continued
• Table 6.2. Comparison of ASO therapy pricings
• Figure 6.1. Key reimbursement and access considerations for emerging gene therapies in AS
• Key insights from the gene therapy market
• Table 6.3. Innovative payer coverage gene therapy insights
• Financial hurdles in AS diagnosis and treatment
• Figure 6.2. Mean annual financial impact on caregivers for AS
• Figure 6.3. Mean annual healthcare costs per patient with development epileptic encephalopathies

• Primary market research approach
• Epidemiology methodology
• Table 7.1. Diagnosed prevalent populations of AS by age

Samples

Companies Mentioned

• Roche
• Ionis
• Biogen
• Ultragenyx
• Neuren Pharmaceuticals
• Foundation for Angelman Syndrome Therapeutics (FAST)
• Encoded Therapeutics
• Transformatx
• Ovid Therapeutics