The gene therapy market is on track to reach $36.55 billion by 2032, driven by increasing demand for innovative treatments targeting genetic disorders at their core. Recent regulatory approvals, including three transformative cell therapies by the FDA in December 2025, signal a pivotal shift in the availability of these advanced medical solutions.
Companies such as Avant Technologies Inc. (OTCQB:AVAI), Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX), CRISPR Therapeutics (NASDAQ:CRSP), Prime Medicine, Inc. (NASDAQ:PRME), and Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL) are at the forefront of this evolving field. The cell and gene therapy sector is projected to expand even further, reaching $39.61 billion by 2034, reflecting a compound annual growth rate of 17.98%.
Breakthroughs in Cell Therapies
Manufacturing advancements in viral vector delivery systems are facilitating the scalable production of gene therapies. This progress is attracting significant investment, with a focus on functional cures rather than ongoing symptom management. Avant Technologies is actively developing cell-based therapies to address diabetes, aging, and chronic diseases through its proprietary encapsulation technology, which shields genetically modified cells from immune rejection.
Avant operates two significant joint ventures aimed at addressing critical health issues. The first, Insulinova, Inc., formed in collaboration with SGAustria Pte. Ltd., is dedicated to creating treatments for type 1 and insulin-dependent type 2 diabetes. By utilizing cell encapsulation technology, Avant aims to enable insulin-producing cells to survive in the body without triggering immune responses, thereby reducing the need for lifelong immunosuppressive medications.
“Cell encapsulation is a game-changer in the field of regenerative medicine,”
stated Chris Winter, CEO of Avant Technologies. He emphasized the potential to enhance patient quality of life by restoring natural glucose control while mitigating risks associated with immune rejection and potential tumor formation.
The diabetes market presents a considerable opportunity, with the International Diabetes Federation estimating that 589 million people are currently living with type 1 and insulin-dependent type 2 diabetes, a figure projected to rise to 853 million by 2050.
Innovative Research and Clinical Trials
Avant’s second joint venture, Klothonova, in partnership with Austrianova, focuses on developing anti-aging therapies that aim to restore circulating α-Klotho levels. Research indicates that decreased α-Klotho levels are linked to serious health issues, including arterial stiffness and vascular calcification. Both joint ventures are based on solid scientific research, supported by over 50 publications that span decades of development.
Vertex Pharmaceuticals is also making strides in this area. At the recent American Society of Hematology Annual Meeting, the company presented promising data on its gene therapy CASGEVY, which showed significant clinical benefits for children aged 5-11 with severe sickle cell disease and transfusion-dependent beta thalassemia. In a Phase 3 study, patients demonstrated a remarkable absence of vaso-occlusive crises for at least 12 months.
Carmen Bozic, M.D., Executive Vice President at Vertex, highlighted the transformative potential of CASGEVY, indicating plans for global regulatory submissions for this age group in the first half of 2026.
Meanwhile, CRISPR Therapeutics shared positive Phase 1 data regarding its investigational gene-editing therapy, CTX310. This therapy targets ANGPTL3, yielding substantial reductions in circulating ANGPTL3 and lipid levels after a single intravenous infusion. The results support the advancement of CTX310 into Phase 1b clinical trials, focusing on severe hypertriglyceridemia.
In the realm of liver diseases, Madrigal Pharmaceuticals has presented compelling two-year data on its product Rezdiffra (resmetirom), demonstrating significant improvements in liver stiffness and fibrosis biomarkers in patients with compensated MASH cirrhosis. David Soergel, M.D., Chief Medical Officer of Madrigal, underscored the urgent need for effective therapies in this area, given the high mortality risk associated with MASH cirrhosis.
Finally, Prime Medicine recently published data demonstrating the safety and efficacy of its investigational product, PM359, aimed at treating chronic granulomatous disease. This therapy has shown promising results in early clinical trials, with patients achieving substantial improvements without significant adverse events.
The gene therapy landscape is rapidly evolving, with industry leaders making substantial strides in research and development. As the market approaches the projected $36.55 billion mark by 2032, the focus remains on innovative solutions that offer hope to patients with previously untreatable genetic disorders.
