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Related Concept Videos

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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Related Experiment Video

Updated: Mar 15, 2026

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
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Assessing the Early Economic Feasibility of a Curative Gene Therapy for Multiple Sclerosis Using a Risk-Adjusted

Attila Imre1,2,3, Balázs Nagy1,2,3, Rok Hren3,4,5

  • 1Center for Health Technology Assessment, Semmelweis University, 1085 Budapest, Hungary.

Healthcare (Basel, Switzerland)
|March 14, 2026
PubMed
Summary

Financial feasibility of IMMUTOL, a novel gene therapy for multiple sclerosis (MS), shows commercial development is unattractive without cost reductions or external support. Current pricing models may not align with the economic realities of advanced therapies.

Keywords:
advanced therapy developmentcost-effectivenessearly-stage health technology assessmentmultiple sclerosisrisk-adjusted net present valuetolerogenic gene therapy

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Area of Science:

  • Biotechnology
  • Gene Therapy
  • Pharmacoeconomics

Background:

  • Multiple sclerosis (MS) presents significant clinical and economic burdens.
  • Existing disease-modifying therapies necessitate lifelong treatment without achieving immune tolerance.
  • IMMUTOL is a novel tolerogenic gene therapy developed to induce durable remission in MS.

Purpose of the Study:

  • To assess the early financial feasibility of IMMUTOL.
  • To evaluate the economic viability of developing a new gene therapy for MS.

Main Methods:

  • Utilized a risk-adjusted net present value (rNPV) model.
  • Incorporated development, operating, and manufacturing costs.
  • Analyzed market dynamics, revenue projections, and probabilities of success.
  • Conducted one-way, probabilistic, and scenario analyses to examine uncertainty.

Main Results:

  • Base-case analysis projected a deterministic rNPV of -$223.8 million.
  • Probabilistic analysis indicated a mean rNPV of -$99.4 million, with 70.2% of simulations yielding negative values.
  • Positive rNPVs were only achieved in scenarios with high treatment prices ($1.5 million) and low manufacturing costs ($200,000).

Conclusions:

  • A structural gap exists between value-based pricing and commercial viability for IMMUTOL.
  • Commercial development appears economically unattractive without significant cost reductions or external financial support.
  • The findings highlight challenges in the economic sustainability of innovative gene therapies for MS.