Category: Gene therapy

In November 2024, the U.S. Food and Drug Administration (FDA) approved Kebilidi (eladocagene exuparvovec-tneq) under accelerated approval, making it the first approved gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. But **what is Kebilidi**, and how does this groundbreaking, one-time treatment offer new hope for those living with this rare and severe neurological disorder?

Antisense oligonucleotides (ASOs) have emerged as a significant therapeutic platform, with several drugs already approved by the FDA for the treatment of rare genetic disorders. This innovative technology provides a way to target the root cause of certain diseases at the RNA level, fundamentally changing how various genetic conditions are treated with ASO.

On November 14, 2024, the FDA granted accelerated approval to eladocagene exuparvovec-tneq (brand name Kebilidi), which was the first brain injected gene therapy approved by the FDA. This milestone marks a new era for treating severe, rare neurological disorders by directly targeting the source of the problem in the central nervous system.

While Zolgensma has been a life-altering gene therapy for infants with Spinal Muscular Atrophy (SMA), experts widely agree that **Why is Zolgensma not a cure?** is a critical question for managing patient expectations. The single-dose treatment halts disease progression but cannot reverse pre-existing neurological damage, a key distinction from a complete cure.

With a reported list price of up to $2.6 million in 2025, Zolgensma therapy is one of the most expensive single-dose treatments in the world. This article explains how much does Zolgensma therapy cost for patients and the healthcare system, exploring the complex factors that influence the final price and outlining available financial assistance.

With an initial price tag exceeding $2.1 million per dose, Zolgensma became known as the world's most expensive medicine upon its approval in 2019. This staggering cost is the result of a complex interplay of factors, including its groundbreaking gene therapy technology, a small patient population, and the innovative pricing strategies employed by pharmaceutical companies.

On December 19, 2017, the FDA approved Luxturna, making history as the first AAV gene therapy for a genetic disease in the U.S.. The therapy, developed by Spark Therapeutics, provides a functional gene to treat inherited retinal dystrophy caused by biallelic mutations in the *RPE65* gene.

Costing $425,000 per eye, Luxturna (voretigene neparvovec-rzyl) is a one-time gene therapy widely recognized as the most expensive eye drug. This revolutionary treatment targets a rare, inherited form of vision loss and represents a significant advancement in the field of ophthalmic medicine.

In clinical trials, a single infusion of valoctocogene roxaparvovec reduced the mean annualized bleeding rate by 83.8% compared to prior Factor VIII prophylaxis [1.10.3]. This one-time gene therapy is transforming treatment, but how is valoctocogene roxaparvovec administered to achieve such significant results?

ONPATTRO (patisiran) was the first-ever small interfering RNA (siRNA) therapeutic approved by the U.S. Food and Drug Administration (FDA) in 2018 for treating a genetic disease. This groundbreaking treatment, which works by "silencing" a gene, has raised a crucial question in the field of genetic medicine: is ONPATTRO a gene therapy?