PTC Therapeutics to Acquire Sangamo Therapeutics’ ST-920 Gene Therapy Program

Shots: PTC Therapeutics has been selected as the winning bidder to acquire ST-920, a BLA-stage, one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics through a competitive bankruptcy auction As per the terms, PTC Therapeutics will pay $111M upfront, with up to $100M in contingent milestone payments tied to certain regulatory approvals; expected to…

ByRidhi Rastogi4 days ago

NEWS

PTC Therapeutics Receives the US FDA’s Approval for Sephience to Treat Phenylketonuria

Shots: The US FDA has approved Sephience (sepiapterin) for the treatment of phenylketonuria (PKU) in pts of all ages, with broad labeling for hyperphenylalaninemia in adults & pediatric pts (≥1mos.) with sepiapterin-responsive PKU; review is ongoing in Japan & Brazil Approval was based on APHENITY study results & long-term extension data showing durable effects and…

ByRidhi RastogiJuly 29, 2025

NEWS

PTC Therapeutics Receives the EC’s Approval for Sephience to Treat Phenylketonuria (PKU)

Shots: The EC has approved Sephience (sepiapterin) to treat PKU in pts of all ages and disease severities across all 30 EEA states; launch is anticipated in Germany in H1 of July Approval was based on APHENITY study results and long-term extension data showing durable effects and improved dietary flexibility Sephience is an oral synthetic…

ByDipanshu DixitJune 24, 2025

NEWS

PTC Therapeutics’ Sephience Receives the CHMP’s Positive Opinion for the Treatment of Phenylketonuria (PKU)

Shots: The CHMP has issued a positive opinion recommending marketing authorization for Sephience (sepiapterin) to treat PKU in patients of all ages and disease severities, expected the EC approval within two mos., with the decision applying across the EU including Iceland, Norway, and Liechtenstein Sepiapterin's NDA is under FDA review (PDUFA: July 29, 2025), and…

ByDipanshu DixitApril 25, 2025

TOP 20

Top 20 Cell and Gene Therapy Companies Based on 2022 Total Revenue

Shots: Standing tall and gradually paving its way through extensive research, Cell & Gene Therapy is a beacon of hope when treating rare and inherited diseases. Cell and gene therapy aims to prevent, treat, and potentially reduce the effects of the underlying cause of genetic diseases. Propelled by constant innovatory winds, the cell and gene…

ByPrince GiriMay 23, 2023

PTC Therapeutics to Acquire Sangamo Therapeutics’ ST-920 Gene Therapy Program

PTC Therapeutics Receives the US FDA’s Approval for Sephience to Treat Phenylketonuria

PTC Therapeutics Receives the EC’s Approval for Sephience to Treat Phenylketonuria (PKU)

PTC Therapeutics’ Sephience Receives the CHMP’s Positive Opinion for the Treatment of Phenylketonuria (PKU)

Top 20 Cell and Gene Therapy Companies Based on 2022 Total Revenue

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