PTC Therapeutics at ASHG 2026

Oct 20–24, 2026 Montréal, Canada ~7.5k attendees Website

PTC Therapeutics at ASHG 2026 in Montréal: 1 presentation (1 poster).

Organization	ASHG 2026 Attendance
PTC Therapeutics South Plainfield, New Jersey	1 Sr. Medical Science Liaison
ptcbio.com\|LinkedIn Public company PTC Therapeutics discovers, develops, and commercializes medicines for children and adults with serious rare diseases. It focuses on neurology and metabolic conditions. Presented data showing patients on its Sephience drug can loosen dietary restrictions while keeping metabolic control, reinforcing its rare-disease position. › Presented data showing patients on its Sephience drug can loosen dietary restrictions while keeping metabolic control, reinforcing its rare-disease position. 2026-08 · source Won a bankruptcy auction to acquire Sangamo's Fabry disease gene-therapy program, expanding its rare-disease and gene-therapy pipeline. 2026-08 · source Reported strong momentum in its Sephience drug launch, signaling continued rare-disease commercial growth. 2026-07 · source Source: company newsroom Q2 2026 revenue $361M, more than double YoY › FY2026 guidance is $1.18-1.28 billion total revenue, driven by the Sephience launch. · 2026-07-30 “Robust Q2 performance with total revenue of $361 million, including $239 million of product revenue” Source: results release	Poster Thu Oct 22 4:15 pm Part of Genetic Therapies and Precision Medicine Poster Thursday Session Diet liberalization and health-related QOL in sepiapterin-treated participants with PKU who were receiving sapropterin at study entry: Interim results from the phase 3 APHENITY extension study Exhibit & Poster Hall · Board 5005T Collaborators: University of California, Los Angeles, University of Colorado Anschutz +3 more Metabolic disorderPharmacologic therapy

Organization

ASHG 2026 Attendance

PTC Therapeutics

South Plainfield, New Jersey

1 Sr. Medical Science Liaison

ptcbio.com|LinkedIn

Public company

PTC Therapeutics discovers, develops, and commercializes medicines for children and adults with serious rare diseases. It focuses on neurology and metabolic conditions.

Presented data showing patients on its Sephience drug can loosen dietary restrictions while keeping metabolic control, reinforcing its rare-disease position.

›

Presented data showing patients on its Sephience drug can loosen dietary restrictions while keeping metabolic control, reinforcing its rare-disease position.

2026-08 · source

Won a bankruptcy auction to acquire Sangamo's Fabry disease gene-therapy program, expanding its rare-disease and gene-therapy pipeline.

2026-08 · source

Reported strong momentum in its Sephience drug launch, signaling continued rare-disease commercial growth.

2026-07 · source

Source: company newsroom

Q2 2026 revenue $361M, more than double YoY

›

FY2026 guidance is $1.18-1.28 billion total revenue, driven by the Sephience launch. · 2026-07-30

“Robust Q2 performance with total revenue of $361 million, including $239 million of product revenue”

Source: results release

Poster

Thu Oct 22
4:15 pm

Part of Genetic Therapies and Precision Medicine Poster Thursday Session

Diet liberalization and health-related QOL in sepiapterin-treated participants with PKU who were receiving sapropterin at study entry: Interim results from the phase 3 APHENITY extension study

Exhibit & Poster Hall · Board 5005T

Collaborators: University of California, Los Angeles, University of Colorado Anschutz +3 more

Metabolic disorderPharmacologic therapy

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