Skysona Global Market Report 2025: Epidemiology, Pipeline Analysis, Market Insights & Forecasts

The skysona market research report is one of a series of new reports that provides skysona market statistics, including skysona industry global market size, regional shares, competitors with a skysona market share, detailed skysona market segments, market trends and opportunities, and any further data you may need to thrive in the skysona industry. This skysona market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

The growth in the forecast period can be attributed to increasing use of real-world evidence, increasing use of telemedicine services, growing insurance coverage options, rising public health initiatives, and increasing demand for medicinal products. Major trends in the forecast period include innovation in pharmaceutical and medical technology, technological development, advanced technologies in manufacturing, a shift towards personalized medicine, and innovations in clinical trials.

The rising prevalence of rare diseases is expected to drive the growth of the Skysona market. Rare diseases are conditions that affect a small percentage of the population, and the increase in their prevalence is due to factors such as improved diagnostic methods, increased awareness among healthcare providers, and advances in genetic testing. Skysona is particularly beneficial for treating rare diseases, such as cerebral adrenoleukodystrophy (CALD), as it provides a gene therapy approach targeting the genetic mutation responsible for the condition. This therapy has the potential to improve neurological outcomes and extend survival for children affected by CALD. For example, in August 2024, ALD Info, a Netherlands-based community initiative, reported that the incidence of adrenoleukodystrophy (ALD) is approximately 1 in 17,000 newborns. A male newborn has a 35-40% risk of developing cerebral ALD between the ages of 3 and 18, although it can also appear in adulthood. As the prevalence of rare diseases increases, the Skysona market is expected to grow.

A significant trend in the Skysona market is the development of innovative gene therapies for rare neurodegenerative disorders. Gene therapies involve modifying a person’s genes to treat or prevent disease. In September 2022, Bluebird Bio Inc., a US-based biotechnology company, received FDA Accelerated Approval for Skysona (elivaldogene autotemcel) to slow the progression of neurologic dysfunction in boys aged 4-17 with early, active cerebral adrenoleukodystrophy (CALD). This one-time gene therapy modifies the patient's own hematopoietic stem cells to produce a functional enzyme, addressing the genetic cause of CALD. The goal is to prevent neurological decline in patients who are in the early stages of the disease, before irreversible damage occurs. This approval marks a major milestone in the treatment of CALD, offering hope for long-term stabilization or improvement of neurological function in affected individuals.

In September 2023, Bluebird Bio, Inc., a biotechnology company based in the US, updated its agreement with Lonza Group AG. Through this collaboration, Lonza intends to enhance its manufacturing capabilities to support the production of Bluebird Bio’s gene therapies, including Zynteglo and Skysona. The goal is to ensure scalable, high-quality manufacturing processes that can meet the growing demand and speed up the delivery of these innovative treatments for rare diseases such as thalassemia and cerebral adrenoleukodystrophy (CALD). Lonza Group AG, based in Switzerland, is a contract manufacturing organization involved in the production of Skysona.

The key company operating in the skysona market is Bluebird bio Inc.

North America was the largest region in the skysona market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in skysona report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the skysona market report are Australia, India, China, South Korea, Japan, UK, France, Germany, USA, Canada.

Skysona is a gene therapy designed to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare genetic disorder that affects the nervous system in young boys. The therapy works by using the patient's own stem cells, which are modified to produce a functional version of the ABCD1 gene. This gene is responsible for producing an enzyme that helps break down fatty acids, and its deficiency leads to the progression of CALD.

Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report’s Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.

The main indication for skysona is cerebral adrenoleukodystrophy (CALD), a rare, genetic neurodegenerative disorder primarily affecting boys. CALD is characterized by the progressive loss of brain function caused by the accumulation of very long-chain fatty acids in the nervous system. Skysona is administered via intravenous infusion. The patient demographics include pediatric and adult patients, and the therapy is distributed through various channels such as hospitals, specialty clinics, and online pharmacies. Key end-users include academic and research institutes, biopharmaceutical companies, and patient advocacy groups.

The skysona market consists of sales of infusion bags, with the dose based on patient weight. Values in this market are ‘factory gate’ values, that is, the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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