Cellular & Gene Therapy in Nevada: A New Era for Rare Disease Treatment
Cellular and gene therapies represent one of the most significant advances in medicine—offering the potential to treat, and in some cases cure, rare diseases at their genetic source. Unlike traditional treatments that manage symptoms, these therapies address the underlying cause of disease, potentially transforming a patient’s health with a single treatment.
For Nevada’s rare disease community, these breakthroughs bring tremendous hope. But they also raise critical questions:
Which therapies are available? Where can patients access them? And what barriers still stand in the way?
What Are Cellular and Gene Therapies?
Gene therapy delivers genetic material into a patient’s cells to replace, inactivate, or introduce genes to treat disease. This can correct genetic defects or provide cells with new instructions to fight disease.
Cell therapy involves transplanting human cells—sometimes genetically modified—to replace or repair damaged tissue. CAR-T cell therapy, for example, reprograms a patient’s own immune cells to attack cancer.
These therapies hold the potential to:
- Address the root genetic cause of disease rather than just managing symptoms
- Reduce long-term healthcare utilization and costs
- Provide durable, potentially lifelong benefits from a single treatment
Current Availability in Nevada
Cellular and gene therapies are increasingly available, though access varies by condition, treatment center, and insurance coverage. Here’s what Nevada patients should know:
FDA-Approved Gene Therapies
Several gene therapies have received FDA approval for rare diseases, including treatments for:
- Sickle cell disease (Casgevy, Lyfgenia)
- Spinal muscular atrophy (Zolgensma)
- Inherited retinal dystrophy (Luxturna)
- Hemophilia A and B (Roctavian, Hemgenix)
- Beta-thalassemia (Zynteglo)
- Cerebral adrenoleukodystrophy (Skysona)
CAR-T Cell Therapies
CAR-T therapies are approved for certain blood cancers and are available at designated treatment centers, including facilities accessible to Nevada patients.
Where Nevada Patients Access Treatment
While many cellular and gene therapies require patients to travel to out-of-state centers of excellence, some treatments are now available right here in Nevada.
Available in Nevada:
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Hemgenix (etranacogene dezaparvovec), a gene therapy for Hemophilia B, is available at Cure 4 The Kids Foundation in Las Vegas through CSL Behring. This represents a significant milestone in bringing cutting-edge gene therapy to Nevada patients.
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Read the official press release to learn more.
Out-of-State Centers:
For therapies not yet available in Nevada, patients commonly travel to:
- California (UCLA, Stanford, UCSF, City of Hope)
- Arizona (Mayo Clinic, Banner Health)
- Utah (Huntsman Cancer Institute, Primary Children’s Hospital)
NV-RDAC is working to expand in-state capacity—building on successes like Hemgenix at C4K—to reduce the burden on families who must travel for care.
Barriers to Access
Despite their promise, significant barriers limit access to cellular and gene therapies for Nevada patients:
Cost: These therapies carry high upfront costs—often ranging from hundreds of thousands to millions of dollars per treatment. While they may reduce long-term healthcare spending, the initial price creates coverage and reimbursement challenges.
Insurance Coverage: Medicaid and private insurers vary widely in their coverage of gene therapies. Prior authorization requirements, coverage denials, and appeals processes can delay or prevent access to treatment.
Geographic Access: While progress is being made—such as Hemgenix availability at Cure 4 The Kids Foundation—Nevada still lacks the specialized infrastructure to administer many gene therapies, requiring some patients to travel out of state for treatment, evaluation, and follow-up care.
Awareness: Many patients, families, and even healthcare providers are unaware of available gene therapies or how to access them.
The CMS Cell and Gene Therapy (CGT) Access Model
In 2024, the Centers for Medicare & Medicaid Services (CMS) launched the CGT Access Model to address some of these barriers for Medicaid recipients.
How the Model Works
The CGT Access Model facilitates outcomes-based agreements between states and pharmaceutical manufacturers. These agreements:
- Lower prices for participating states
- Tie payment to actual treatment outcomes
- Reduce financial risk for state Medicaid programs
Current Focus: Sickle Cell Disease
The model initially focuses on gene therapy for sickle cell disease—a genetic blood disorder that disproportionately affects Black Americans and has historically been associated with poor health outcomes, limited treatment options, and significant health disparities.
By increasing access to transformative therapies, the model aims to address these longstanding inequities.
Nevada’s Opportunity
- The CGT Access Model is voluntary for states and manufacturers
- States can participate anytime between January 2025 and January 2026
- Funding is available through a Notice of Funding Opportunity (NOFO), though it’s not required for participation
NV-RDAC is actively monitoring this model and advocating for Nevada’s participation to expand Medicaid access to gene therapies for eligible patients.
What NV-RDAC Is Doing
The Nevada Rare Disease Advisory Council is committed to expanding access to cellular and gene therapies for all Nevadans. Our efforts include:
Advocacy for Medicaid Coverage
- Working with the Nevada Health Authority (NVHA) to address coverage gaps for gene therapies
- Supporting Nevada’s potential participation in the CMS CGT Access Model
- Advocating for reimbursement policies that reflect the complexity and value of these treatments
Reducing Geographic Barriers
- Celebrating and building on milestones like Hemgenix availability at Cure 4 The Kids Foundation
- Identifying opportunities to bring additional gene therapies to Nevada
- Supporting telehealth expansion for pre-treatment evaluation and post-treatment follow-up
- Advocating for travel assistance programs for families who must seek treatment out of state
Raising Awareness
- Educating healthcare providers about available gene therapies and referral pathways
- Connecting patients and families with resources and information about treatment options
- Partnering with disease-specific organizations to ensure patients know what’s available
Data Collection
- Tracking gene therapy access and outcomes through our partnership with Cure 4 The Kids Foundation
- Using needs assessment data to document barriers and inform policy recommendations
Resources for Patients and Families
If you or a loved one is considering cellular or gene therapy, these resources can help:
Finding Treatment Centers
- FDA Approved Cellular and Gene Therapy Products
- Disease-specific foundations often maintain lists of treatment centers
Understanding Coverage
- Contact your insurance provider to understand coverage and prior authorization requirements
- Nevada Medicaid
- For Medicaid patients: Ask about the CMS CGT Access Model
Financial Assistance
- Many manufacturers offer patient assistance programs for approved therapies
- Disease-specific foundations may offer financial support for treatment-related costs
Looking Ahead
Cellular and gene therapies are no longer a distant promise—they are here, and more are coming. As new therapies receive FDA approval and coverage policies evolve, NV-RDAC will continue advocating for Nevada patients to have equitable access to these life-changing treatments.
No Nevadan should be denied a potential cure because of where they live, what insurance they have, or what they can afford. That’s the future we’re working toward.
For questions about cellular and gene therapy access in Nevada, contact NV-RDAC or attend one of our regularly scheduled meetings.