Gene therapy Market Key Highlights and Future Opportunities Till 2035

The global gene therapy market is estimated to grow from $2.4 billion in 2024 to $16.5 billion by 2035, representing a CAGR of 19.25% during the forecast period till 2035.

The gene therapy market landscape has transformed significantly since the approval of the world’s first gene therapy, Gendicine®, in the early 2000’s. Ever since then, with the constant R&D efforts of the gene therapy companies and non-industry stakeholder, an increasing number of therapies have entered the gene therapy market. It is worth highlighting that, more than 390 gene therapies have either received regulatory approval or are currently being evaluated in across various phases of clinical trials to assess the safety and effectiveness of these medications in patients diagnosed with different types of inherited disorders. In fact, close to 5% of the gene therapies have already been marketed, of which 30% of the therapies are intended for the treatment of various hematological disorders that are caused due to abnormalities in specific genes of an individual. Additionally, at present, 920 gene therapies are either in discovery stage or being evaluated in preclinical studies, of which 60% of the therapies are being developed to be delivered using adeno-associated virus vectors. Further, during our research, we identified 345 gene therapy developers / gene therapy companies that are engaged in the development of gene therapies.
The majority (58%) of gene therapy developers were established during the period 2011-2020. This period has seen a notable increase in the number of players entering the gene therapy domain, reflecting a stable and enticing industry for the new entrants. In fact, the positive clinical outcomes demonstrated by these therapies in real-world scenarios have attracted several start-ups to enter the domain. As such, it is worth mentioning that since 2020, close to 30 developers have entered the market, indicating the growing interest of stakeholders in this field. Notable examples of such players (in reverse chronological order) include Addimmune (2023), Stemcardia (2023), Celosia Therapeutics (2022), Theriva Biologics (2022), Borea Therapeutics (2021), Gromit Therapeutics (2021) and Ray Therapeutics (2021).
Close to 80% of the marketed and clinical stage gene therapies have received orphan drug designation. This can be attributed to the fact that around 80% of rare diseases worldwide are genetic in nature. Hence, in order to overcome the burden associated with these conditions, regulatory bodies have proactively granted orphan drug designations (granted to drugs developed for the treatment of rare diseases ) to gene therapies that have demonstrated clinical efficacy in addressing the underlying genetic cause of such orphan indications. This is followed by gene therapies that have received fast track designation (48%) and rare pediatric disease designation (31%).
Further, close to 90% of marketed and clinical stage gene therapies are delivered using the in vivo method primarily due to the fact that in vivo gene delivery is typically a simpler process, which involves direct administration of genetic material to the target organ within the body. Moreover, unlike ex vivo gene therapies, in vivo gene delivery is a less-invasive process, as it minimizes the need for procedures such as cell extraction and in vitro genetic modification; thereby maximizing the patient compliance to these gene therapies. Further, over 10% of the gene therapies that are currently being developed are delivered using ex vivo methods. Examples of marketed gene therapies delivered through ex vivo method (arranged in alphabetical order) includes Casgevy™ (CRISPR Therapeutics and Vertex Pharmaceuticals), INVOSSA® (Kolon TissueGene) and Libmeldy™ (Orchard Therapeutics and SR-Tiget).
Majority (60%) of preclinical and discovery stage gene therapies are delivered using adeno-associated virus vectors. This is followed by therapies delivered using non-viral vectors (14%) and lentivirus vectors (7%). Notable examples of gene therapies delivered using non-viral vectors (arranged in alphabetical order, being developed for treatment of genetic disorders) include CS-104 (CorrectSequence Therapeutics), EG-i08 (enGene), GF-1003 (Genflow Biosciences) and RY107 (Reyon Pharmaceutical and MDimune).
Further, a maximum (23%) of the preclinical and discovery stage gene therapies are being developed for the treatment of neurological disorders. It is important to note that neurological disorders are currently the second leading cause of death worldwide. In fact, according to the World Federation of Neurology, in 2023, over 40% of the global population had suffered from some form of neurological conditions and these statistics are likely to double by 2050. As a result, there is an alarming need to develop effective therapies that can target the root cause of these diseases and can address the current unmet needs. In this regard, gene therapies have emerged as a promising therapeutic modality as they are designed to cross the blood-brain barrier and deliver functional genes, potentially treating a range of inherited neurological disorders.
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