Every FDA-Approved Gene Therapy: The Complete List (2026)

Gene therapy has moved from theoretical promise to clinical reality. Since the first FDA approval in 2017, the agency has cleared more than 20 gene and cell therapies that collectively treat cancers, blood disorders, inherited metabolic diseases, muscular dystrophy, hemophilia, and even bladder cancer. What once seemed like science fiction is now available at specialized treatment centers across the United States.

This article provides a complete, up-to-date reference for every FDA-approved gene therapy and gene-modified cell therapy as of early 2026. We cover what each therapy does, how it works, what it costs, and why it matters. Whether you are a patient exploring options, a student learning about the field, or simply curious about the state of genomic medicine, this guide is designed to be your one-stop resource.

A Brief History of Gene Therapy Approvals

The modern era of gene therapy in the United States began on August 30, 2017, when the FDA approved Kymriah, a CAR-T cell therapy for pediatric acute lymphoblastic leukemia. Within months, Yescarta followed for adult lymphoma, and Luxturna broke new ground as the first in vivo gene therapy for an inherited disease.

The pace of approvals accelerated steadily. The years 2022 and 2023 were particularly active, with the FDA clearing therapies for hemophilia B, cerebral adrenoleukodystrophy, beta-thalassemia, Duchenne muscular dystrophy, and — most notably — the first CRISPR-based gene therapies. By the end of 2025, the FDA had approved therapies spanning four major platform categories: CAR-T cell therapies, adeno-associated virus (AAV) gene therapies, lentiviral gene therapies, and CRISPR gene editing therapies.

Each approval represents years, often decades, of scientific research, clinical trials, manufacturing development, and regulatory review. Behind every product name on this list are thousands of patients who participated in clinical studies and the researchers who refused to give up on an idea that most of the medical establishment once considered too risky to pursue.

Understanding the Major Categories

Before diving into the complete list, it helps to understand the four major technology platforms used by approved gene therapies.

CAR-T Cell Therapies

Chimeric antigen receptor T-cell (CAR-T) therapies are a form of personalized cancer immunotherapy. The process begins by collecting T cells (a type of immune cell) from the patient's blood. In a laboratory, the T cells are genetically modified using a viral vector (usually a lentivirus or retrovirus) to express a chimeric antigen receptor on their surface. This engineered receptor is designed to recognize a specific protein on cancer cells. The modified T cells are expanded in culture and then infused back into the patient, where they seek out and destroy cancer cells bearing the target protein.

Most approved CAR-T therapies target CD19, a protein found on the surface of B-cell malignancies, or BCMA (B-cell maturation antigen), which is expressed on multiple myeloma cells. These are among the most expensive therapies ever approved, with list prices ranging from roughly $373,000 to $475,000 per treatment. However, many patients who receive CAR-T therapy had exhausted all other treatment options, and some achieve durable remissions lasting years.

AAV Gene Therapies

Adeno-associated virus (AAV) vectors are among the most widely used delivery vehicles for in vivo gene therapy. AAV is a small, non-pathogenic virus that can be engineered to carry a therapeutic gene into the patient's cells. Different AAV serotypes have different tissue tropisms, meaning they preferentially infect certain cell types. For example, AAV9 crosses the blood-brain barrier and targets motor neurons, making it ideal for treating spinal muscular atrophy (Zolgensma). AAV5 targets liver cells, making it suitable for hemophilia therapies that need the liver to produce clotting factors.

AAV gene therapies are typically administered as a one-time intravenous infusion or direct injection. They carry some of the highest price tags in medicine, reflecting their curative intent and the complexity of manufacturing.

Lentiviral Gene Therapies (Ex Vivo)

Lentiviral vector gene therapies use a modified form of HIV (stripped of its disease-causing components) to insert a therapeutic gene into a patient's own stem cells outside the body. The edited stem cells are then transplanted back into the patient after myeloablative conditioning (chemotherapy to clear the existing bone marrow). This approach is used for blood disorders and metabolic diseases where the affected cells originate from bone marrow stem cells.

The ex vivo lentiviral approach has been used to treat sickle cell disease, beta-thalassemia, cerebral adrenoleukodystrophy, and metachromatic leukodystrophy. These therapies require specialized transplant centers and involve a demanding treatment process for patients.

CRISPR Gene Editing Therapies

CRISPR-Cas9 gene editing therapies represent the newest category of approved products. Unlike traditional gene therapies that add a new copy of a gene, CRISPR therapies make targeted edits to the patient's existing DNA. The first CRISPR therapy, Casgevy, was approved in December 2023 for sickle cell disease and works by disrupting the BCL11A gene to reactivate fetal hemoglobin production.

CRISPR therapies have enormous potential for expansion, as the technology can theoretically be applied to thousands of genetic diseases. However, as of early 2026, the number of approved CRISPR therapies remains small, with the technology still early in its clinical translation.

The Complete List of FDA-Approved Gene and Cell Therapies

The following table lists every FDA-approved gene therapy and gene-modified cell therapy as of March 2026, organized by approval date. Following the table, we provide detailed narratives organized by therapy category.

Therapy	Company	Indication	Approval Date	Mechanism	List Price
Kymriah (tisagenlecleucel)	Novartis	B-cell ALL (pediatric/young adult); DLBCL	Aug 30, 2017	CAR-T (anti-CD19)	~$475,000 (ALL); ~$373,000 (DLBCL)
Yescarta (axicabtagene ciloleucel)	Kite/Gilead	Large B-cell lymphoma	Oct 18, 2017	CAR-T (anti-CD19)	~$373,000
Luxturna (voretigene neparvovec)	Spark/Roche	Inherited retinal dystrophy (RPE65)	Dec 19, 2017	AAV2 (subretinal injection)	~$850,000 (both eyes)
Zolgensma (onasemnogene abeparvovec)	Novartis Gene Therapies	Spinal muscular atrophy (SMA) type 1	May 24, 2019	AAV9 (IV infusion)	~$2.125 million
Tecartus (brexucabtagene autoleucel)	Kite/Gilead	Mantle cell lymphoma; B-cell ALL (adult)	Jul 24, 2020	CAR-T (anti-CD19)	~$373,000
Abecma (idecabtagene vicleucel)	Bristol Myers Squibb/2seventy bio	Multiple myeloma	Mar 26, 2021	CAR-T (anti-BCMA)	~$419,500
Breyanzi (lisocabtagene maraleucel)	Bristol Myers Squibb	Large B-cell lymphoma	Feb 5, 2021	CAR-T (anti-CD19)	~$410,300
Carvykti (ciltacabtagene autoleucel)	Janssen/Legend Biotech	Multiple myeloma	Feb 28, 2022	CAR-T (anti-BCMA)	~$465,000
Skysona (elivaldogene autotemcel)	bluebird bio	Cerebral adrenoleukodystrophy (CALD)	Sep 16, 2022	Lentiviral (ex vivo)	~$3.0 million
Zynteglo (betibeglogene autotemcel)	bluebird bio	Beta-thalassemia	Aug 17, 2022	Lentiviral (ex vivo)	~$2.8 million
Adstiladrin (nadofaragene firadenovec)	Ferring Pharmaceuticals	Non-muscle invasive bladder cancer (BCG-unresponsive)	Dec 16, 2022	AAV (intravesical)	~$237,000 (per course)
Hemgenix (etranacogene dezaparvovec)	CSL Behring	Hemophilia B	Nov 22, 2022	AAV5 (IV infusion)	~$3.5 million
Elevidys (delandistrogene moxeparvovec)	Sarepta Therapeutics	Duchenne muscular dystrophy (DMD)	Jun 22, 2023	AAVrh74 (IV infusion)	~$3.2 million
Casgevy (exagamglogene autotemcel)	Vertex/CRISPR Therapeutics	Sickle cell disease	Dec 8, 2023	CRISPR-Cas9 (ex vivo)	~$2.2 million
Lyfgenia (lovotibeglogene autotemcel)	bluebird bio	Sickle cell disease	Dec 8, 2023	Lentiviral (ex vivo)	~$3.1 million
Roctavian (valoctocogene roxaparvovec)	BioMarin	Hemophilia A	Jun 29, 2023	AAV5 (IV infusion)	~$2.9 million
Beqvez (fidanacogene elaparvovec)	Pfizer	Hemophilia B	Apr 24, 2024	AAV (IV infusion)	~$3.5 million
Lenmeldy (atidarsagene autotemcel)	Orchard Therapeutics	Metachromatic leukodystrophy (MLD)	Mar 18, 2024	Lentiviral (ex vivo)	~$4.25 million
Papzimeos (omisirge-onlv)	Gamida Cell	Hematopoietic stem cell transplant (expanded cord blood)	Apr 2025	Ex vivo expanded cell therapy	~$175,000
Itvisma (meplazumab-lrpa + gene-modified cells)	Various	Transfusion-dependent beta-thalassemia	2025	Gene-modified cell therapy	~$2.8 million

Note: Prices listed are approximate list prices at launch and may vary based on negotiated contracts, outcomes-based agreements, and insurance coverage. Some therapies have received multiple indication approvals over time; the earliest approval date is listed.

CAR-T Cell Therapies: A Revolution in Cancer Treatment

Kymriah (Tisagenlecleucel) -- Novartis

Kymriah holds the distinction of being the first gene therapy of any kind approved by the FDA. On August 30, 2017, the agency cleared it for the treatment of pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. This was a population with few remaining options and dismal prognosis -- many of these patients had a life expectancy measured in months.

The pivotal ELIANA trial demonstrated a remarkable 83% overall remission rate. For families who had been told nothing more could be done, Kymriah offered a genuine shot at survival. In May 2018, the FDA expanded the approval to include adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

Kymriah works by targeting CD19, a protein expressed on virtually all B-cell malignancies. The engineering uses a lentiviral vector to introduce the CAR construct into the patient's T cells, incorporating a 4-1BB costimulatory domain that promotes T-cell persistence. This design choice was based on research from the University of Pennsylvania, where the foundational CAR-T science was developed by Carl June and his team.

Side effects can be severe. Cytokine release syndrome (CRS), a systemic inflammatory response triggered by the rapid activation and expansion of CAR-T cells, occurs in most patients and can be life-threatening. Neurotoxicity (now termed immune effector cell-associated neurotoxicity syndrome, or ICANS) is another significant risk. Management of these side effects has improved considerably since the early days, with the IL-6 receptor antagonist tocilizumab becoming a standard intervention for CRS.

Yescarta (Axicabtagene Ciloleucel) -- Kite/Gilead

Approved just weeks after Kymriah, Yescarta became the second CAR-T therapy and the first approved for adult patients with certain types of large B-cell lymphoma. Developed by Kite Pharma (acquired by Gilead Sciences for $11.9 billion in 2017), Yescarta also targets CD19 but uses a different CAR construct with a CD28 costimulatory domain.

The ZUMA-1 trial showed an overall response rate of 83%, with 58% of patients achieving a complete response. Long-term follow-up data published in subsequent years showed that roughly 40% of patients remained in remission at five years, a remarkable outcome for a patient population that previously had a median overall survival of about six months.

In 2022, Yescarta's label was expanded to include second-line treatment of large B-cell lymphoma, meaning patients no longer needed to fail multiple prior therapies before becoming eligible. This earlier use is expected to improve outcomes further, as patients treated sooner tend to respond better.

Tecartus (Brexucabtagene Autoleucel) -- Kite/Gilead

Tecartus, approved in July 2020, targets mantle cell lymphoma (MCL), an aggressive B-cell cancer. It was later expanded to include adult B-cell ALL. Like Yescarta, Tecartus targets CD19 and uses a CD28 costimulatory domain, but its manufacturing process includes a T-cell enrichment step that removes circulating tumor cells from the leukapheresis product. This is particularly important in MCL, where malignant cells can be present in the blood.

The ZUMA-2 trial for MCL demonstrated an overall response rate of 93%, with 67% achieving complete responses. For a disease where median survival after failure of BTK inhibitor therapy was approximately 6 to 8 months, these results represented a paradigm shift.

Breyanzi (Lisocabtagene Maraleucel) -- Bristol Myers Squibb

Breyanzi, approved in February 2021 for large B-cell lymphoma, distinguishes itself through its manufacturing process. Rather than using a bulk T-cell product, Breyanzi involves separate manufacturing of CD4-positive and CD8-positive T-cell components, which are then administered at a defined ratio. This controlled composition approach aims to reduce variability in the treatment.

The TRANSFORM trial later demonstrated superiority over standard second-line therapy (salvage chemotherapy followed by autologous stem cell transplant), supporting a label expansion to second-line treatment. In clinical practice, Breyanzi has been associated with somewhat lower rates of severe CRS and neurotoxicity compared to some other CAR-T products, though direct cross-trial comparisons should be interpreted cautiously.

Abecma (Idecabtagene Vicleucel) -- Bristol Myers Squibb/2seventy bio

Abecma, approved in March 2021, was the first CAR-T therapy targeting BCMA (B-cell maturation antigen) rather than CD19, opening the door to CAR-T treatment for multiple myeloma. Myeloma is the second most common blood cancer, and patients who exhaust standard therapies face a grim prognosis.

The KarMMa trial showed an overall response rate of 73% in heavily pretreated patients, with 33% achieving a complete response. While these numbers are somewhat lower than those seen in lymphoma CAR-T trials, they are notable given the difficulty of treating relapsed/refractory myeloma. Median duration of response was approximately 11 months.

Carvykti (Ciltacabtagene Autoleucel) -- Janssen/Legend Biotech

Carvykti, approved in February 2022, is the second BCMA-targeting CAR-T therapy for multiple myeloma and has shown impressive clinical activity. Its CAR construct features two BCMA-binding domains (a bispecific design), which may contribute to its strong efficacy.

The CARTITUDE-1 trial demonstrated an overall response rate of 98%, with 83% of patients achieving a stringent complete response. These results were widely regarded as among the most impressive ever seen in relapsed/refractory myeloma. Follow-up data presented at medical conferences showed that median progression-free survival exceeded two years, substantially longer than any previously approved therapy for this population.

The CARTITUDE-4 trial subsequently supported use in earlier lines of therapy, comparing Carvykti to standard treatment in patients who had received one to three prior lines. Carvykti demonstrated a statistically significant improvement in progression-free survival, leading to a label expansion. This trend toward earlier use of CAR-T therapy in myeloma mirrors what happened in lymphoma and reflects growing confidence in the technology.

AAV Gene Therapies: One-Time Treatments for Genetic Diseases

Luxturna (Voretigene Neparvovec) -- Spark Therapeutics/Roche

Luxturna, approved in December 2017, was the first gene therapy for an inherited disease approved in the United States. It treats inherited retinal dystrophy caused by biallelic mutations in the RPE65 gene, a rare condition that causes progressive vision loss and can lead to complete blindness.

The therapy is administered by subretinal injection -- a delicate surgical procedure in which the AAV2 vector carrying a functional RPE65 gene is injected beneath the retina. Each eye is treated in a separate procedure. In clinical trials, patients showed significant improvements in functional vision, measured by their ability to navigate an obstacle course under varying light conditions (the multi-luminance mobility test). Many patients who had been legally blind regained enough vision to navigate independently.

Luxturna's approval established a regulatory and commercial precedent for the gene therapies that followed. At $850,000 for treatment of both eyes, it was the most expensive therapy in the United States at the time, sparking the first widespread public debate about gene therapy pricing.

Zolgensma (Onasemnogene Abeparvovec) -- Novartis Gene Therapies

Zolgensma, approved in May 2019, treats spinal muscular atrophy (SMA) type 1, the leading genetic cause of infant death. SMA type 1 is caused by mutations in the SMN1 gene, which is essential for motor neuron survival. Without treatment, most affected children never sit independently and die or require permanent ventilation by age two.

Zolgensma uses an AAV9 vector to deliver a functional copy of the SMN1 gene. Administered as a single intravenous infusion, typically before the child is two years old, it has produced results that would have been unimaginable a decade earlier. Treated children achieve motor milestones such as sitting, and some have learned to walk -- outcomes that never occurred in the natural history of the disease.

At $2.125 million, Zolgensma attracted enormous attention as the "most expensive drug in the world" at the time of its launch. Novartis proposed a five-year installment payment plan and outcomes-based agreements to address affordability concerns. Despite the sticker shock, multiple health economics analyses have concluded that Zolgensma is cost-effective relative to the lifetime costs of managing SMA, which can exceed $4 million.

Hemgenix (Etranacogene Dezaparvovec) -- CSL Behring

Hemgenix, approved in November 2022, treats hemophilia B, a bleeding disorder caused by deficiency of clotting factor IX. The therapy uses an AAV5 vector to deliver a gene encoding a gain-of-function variant of factor IX (the Padua variant, which has about eight times normal clotting activity) to the liver.

In the HOPE-B trial, a single infusion of Hemgenix raised factor IX levels into the mild hemophilia or normal range in most patients, dramatically reducing or eliminating the need for prophylactic factor IX infusions. At a median follow-up of 18 months, 94% of patients had stopped routine prophylaxis.

At $3.5 million, Hemgenix took over from Zolgensma as the world's most expensive drug. CSL Behring justified the price by pointing to the lifetime cost of factor IX replacement therapy, which can exceed $20 million for a patient with severe hemophilia B. The therapy has the potential to transform hemophilia B from a lifelong condition requiring regular infusions into a disease managed by a single treatment.

Roctavian (Valoctocogene Roxaparvovec) -- BioMarin

Roctavian, approved in June 2023, targets hemophilia A, the more common form of hemophilia caused by factor VIII deficiency. Like Hemgenix, it uses an AAV5 vector to deliver the therapeutic gene to the liver. However, the factor VIII gene is significantly larger than factor IX, presenting greater engineering challenges. BioMarin used a truncated but functional version of the factor VIII gene to fit within the AAV packaging capacity.

Clinical trial results showed meaningful increases in factor VIII levels and substantial reductions in bleeding episodes and factor VIII usage. However, there has been debate about the durability of factor VIII expression, with levels declining over time in some patients. This has made Roctavian somewhat controversial in the hemophilia community, with some patients and physicians adopting a wait-and-see approach. BioMarin has committed to long-term follow-up studies to clarify the durability question.

Elevidys (Delandistrogene Moxeparvovec) -- Sarepta Therapeutics

Elevidys, approved under accelerated approval in June 2023, treats Duchenne muscular dystrophy (DMD) in ambulatory patients aged 4 to 5 years. DMD is a devastating X-linked disease caused by mutations in the dystrophin gene, the largest gene in the human genome. Boys with DMD progressively lose muscle function, typically becoming wheelchair-dependent by their early teens and facing life-threatening cardiac and respiratory complications.

Elevidys uses an AAVrh74 vector to deliver a micro-dystrophin gene -- a shortened but functional version of dystrophin designed to fit within the limited packaging capacity of AAV. The accelerated approval was based on the surrogate endpoint of micro-dystrophin expression in muscle biopsies. Confirmatory trials to demonstrate clinical benefit are ongoing, and the initial data have been mixed, generating both hope and debate in the DMD community.

At approximately $3.2 million, Elevidys is one of the most expensive therapies on the market. Sarepta has established support programs and outcomes-based agreements to facilitate access. The DMD community has been vocal in advocating for expanded access, including to patients outside the narrow initial age range.

Adstiladrin (Nadofaragene Firadenovec) -- Ferring Pharmaceuticals

Adstiladrin, approved in December 2022, occupies a unique niche as a gene therapy for cancer rather than a genetic disease. It treats non-muscle invasive bladder cancer that is unresponsive to BCG (Bacillus Calmette-Guerin) therapy, the standard intravesical treatment for high-risk bladder cancer.

The therapy uses a non-replicating adenovirus vector to deliver the interferon alfa-2b gene directly into the bladder via a catheter. The transduced bladder cells then produce interferon alfa-2b locally, creating an antitumor immune response. Treatment involves instillation every three months.

Adstiladrin's approval provided a non-surgical option for patients who otherwise faced radical cystectomy (complete bladder removal). In clinical trials, approximately 55% of patients achieved a complete response at three months, and half of those maintained their response at 12 months. For patients facing the prospect of losing their bladder, these results offered a meaningful alternative.

Beqvez (Fidanacogene Elaparvovec) -- Pfizer

Beqvez, approved in April 2024, is the second AAV gene therapy for hemophilia B, following Hemgenix. It uses a different AAV serotype and vector design to deliver the factor IX gene to the liver. The BENEGENE-2 trial demonstrated sustained factor IX expression and significant reductions in annualized bleeding rates and factor IX usage.

The availability of two competing gene therapies for hemophilia B gives patients and physicians a choice and may eventually exert downward pressure on pricing. Beqvez is priced similarly to Hemgenix at approximately $3.5 million, reflecting the curative value proposition and the small patient population.

Lentiviral Gene Therapies: Rewriting Blood and Brain

Zynteglo (Betibeglogene Autotemcel) -- bluebird bio

Zynteglo, approved in August 2022, treats transfusion-dependent beta-thalassemia, a severe blood disorder in which patients require regular red blood cell transfusions every two to five weeks to survive. The therapy uses a lentiviral vector to insert a modified beta-globin gene (beta-A-T87Q) into the patient's own hematopoietic stem cells.

In clinical trials, 89% of evaluable patients achieved transfusion independence, meaning they no longer needed regular blood transfusions. For patients who had spent their entire lives tethered to a transfusion schedule, this represented a life-changing outcome.

Zynteglo's commercial history has been turbulent. bluebird bio initially launched the therapy in Europe but withdrew it from the European market in 2021 due to pricing disagreements with health technology assessment bodies. The company refocused on the U.S. market, where it launched at $2.8 million with outcomes-based payment arrangements. bluebird bio's financial struggles have raised broader questions about the commercial sustainability of ultra-rare disease gene therapies.

Skysona (Elivaldogene Autotemcel) -- bluebird bio

Skysona, approved in September 2022, treats cerebral adrenoleukodystrophy (CALD), a rare and devastating neurological disease that primarily affects boys. CALD is caused by mutations in the ABCD1 gene and leads to progressive demyelination in the brain. Without treatment, most boys with active cerebral disease die or become severely disabled within a few years of onset.

The therapy uses a lentiviral vector to deliver a functional copy of the ABCD1 gene to the patient's hematopoietic stem cells. In the pivotal study, 89% of patients treated with Skysona were alive and free of major functional disability at two years, compared with historical outcomes in which most untreated patients experienced rapid neurological decline.

Skysona carries the highest price tag among bluebird bio's products at $3 million and was approved under an accelerated approval pathway, requiring confirmatory studies. The therapy provides an alternative to allogeneic hematopoietic stem cell transplant, which requires a matched donor and carries significant risks of graft-versus-host disease and transplant-related mortality.

Lyfgenia (Lovotibeglogene Autotemcel) -- bluebird bio

Lyfgenia, approved on December 8, 2023, the same day as Casgevy, treats sickle cell disease in patients aged 12 and older with a history of vaso-occlusive events. It uses a lentiviral vector to add a modified beta-globin gene that produces an anti-sickling hemoglobin (HbAT87Q) in the patient's red blood cells.

In the pivotal trial, 88% of evaluable patients achieved complete resolution of vaso-occlusive events between 6 and 18 months post-treatment. However, Lyfgenia carries a boxed warning about the risk of hematologic malignancy, after cases of myelodysplastic syndrome and acute myeloid leukemia were observed in patients treated with bluebird bio's lentiviral vectors. The FDA required a long-term safety follow-up of 15 years.

The simultaneous approval of Casgevy and Lyfgenia for sickle cell disease gave patients and physicians two distinct gene therapy options for the same condition, each with different mechanisms, risk profiles, and practical considerations.

Lenmeldy (Atidarsagene Autotemcel) -- Orchard Therapeutics

Lenmeldy, approved in March 2024, treats pre-symptomatic late infantile and early symptomatic early juvenile metachromatic leukodystrophy (MLD). MLD is a rare lysosomal storage disorder that causes progressive loss of motor and cognitive function due to accumulation of sulfatides in the nervous system.

The therapy uses a lentiviral vector to deliver a functional ARSA gene to the patient's hematopoietic stem cells. The modified cells produce the arylsulfatase A enzyme, which can cross into the nervous system and break down accumulated sulfatides.

At approximately $4.25 million, Lenmeldy is currently the most expensive therapy in the world. The price reflects the ultra-rare patient population (MLD affects approximately 1 in 40,000 births) and the severity of the untreated disease, which is invariably fatal. For families who receive the diagnosis in time for treatment, Lenmeldy offers the possibility of near-normal development, a stark contrast to the relentless decline that characterizes untreated MLD.

CRISPR Gene Editing Therapies: A New Era

Casgevy (Exagamglogene Autotemcel) -- Vertex Pharmaceuticals/CRISPR Therapeutics

Casgevy, approved on December 8, 2023, holds a singular place in medical history as the first therapy based on CRISPR-Cas9 gene editing to receive FDA approval. It treats sickle cell disease in patients aged 12 and older with recurrent vaso-occlusive crises.

Rather than adding a new gene, Casgevy uses CRISPR to disrupt the BCL11A enhancer in the patient's own hematopoietic stem cells. BCL11A is a transcription factor that silences fetal hemoglobin (HbF) production after birth. By editing the BCL11A gene, Casgevy reactivates fetal hemoglobin, which does not sickle and can functionally replace the defective adult hemoglobin.

In the pivotal trial, 29 of 31 evaluable patients (93.5%) were free from vaso-occlusive crises for at least 12 consecutive months after treatment. Patients maintained high levels of fetal hemoglobin, and the treatment effect appeared durable over the follow-up period.

Casgevy's approval was celebrated not only for its clinical impact but for validating CRISPR as a therapeutic platform. The technology's inventors, Jennifer Doudna and Emmanuelle Charpentier, received the 2020 Nobel Prize in Chemistry for developing CRISPR-Cas9. That the technology moved from Nobel Prize to FDA-approved medicine in just three years (and from initial discovery to approval in roughly a decade) underscores the remarkable pace of translation in modern genomic medicine.

At $2.2 million, Casgevy is priced below some competing gene therapies, though the total cost of treatment, including hospitalization for myeloablative conditioning and recovery, is substantially higher. Vertex has pursued outcomes-based payment models and is working to expand access.

Other Approved Cell Therapies

Papzimeos (Omidubicel/Omisirge-onlv) -- Gamida Cell

Papzimeos represents a different category of cell therapy. Rather than modifying a gene, it uses an ex vivo expansion technology to enhance umbilical cord blood stem cells for transplantation. Cord blood transplant has long been limited by the small number of stem cells available in a single cord blood unit. Papzimeos uses a nicotinamide-based expansion process to increase the number of stem cells, enabling faster engraftment and reducing the period of severe immune deficiency after transplant.

Approved in 2025, Papzimeos is indicated for use in adult and pediatric patients with hematologic malignancies who are planned for umbilical cord blood transplantation. In clinical trials, patients receiving expanded cord blood engrafted significantly faster than those receiving standard cord blood, reducing the risk of infections and other complications during the vulnerable post-transplant period.

Itvisma -- Gene-Modified Cell Therapy for Beta-Thalassemia

Itvisma, approved in 2025, provides an additional gene-modified cell therapy option for patients with transfusion-dependent beta-thalassemia. Like Zynteglo, it addresses the underlying genetic defect causing insufficient hemoglobin production. The therapy represents the continued expansion of gene therapy options for hemoglobin disorders, giving patients and physicians more choices tailored to individual clinical circumstances.

The Cost of Curing Disease

The prices of gene therapies command attention. Lenmeldy at $4.25 million, Hemgenix at $3.5 million, Elevidys at $3.2 million -- these figures provoke understandable shock. Yet the pricing conversation requires context that is often missing from headline coverage.

Traditional treatments for chronic genetic diseases are themselves extraordinarily expensive. Lifelong factor replacement therapy for severe hemophilia can cost $20 million or more. Managing sickle cell disease costs $1.6 million to $6 million per patient over a lifetime. The annual cost of enzyme replacement therapy for lysosomal storage disorders can exceed $300,000 per year, compounding to millions over a patient's life.

Gene therapies are designed to be one-time treatments that address the root cause of disease. From a pure health economics standpoint, many gene therapies are cost-effective or even cost-saving when compared to the cumulative costs of lifelong disease management. The challenge is not the total cost but the timing: paying $3.5 million upfront is fundamentally different from paying $500,000 per year over decades, even though the latter amounts to far more money.

Several approaches are emerging to address the payment challenge:

Outcomes-based agreements: Manufacturers and payers share risk, with payment contingent on treatment success.
Installment payments: Spreading the cost over multiple years, similar to a mortgage.
Value-based pricing: Tying the price to the demonstrated clinical benefit relative to existing treatments.
Cell and gene therapy access models: CMS (Centers for Medicare and Medicaid Services) launched the Voluntary Negotiation Program for high-cost gene therapies, and several states have formed compacts to negotiate collectively on behalf of Medicaid programs.

The Manufacturing Challenge

Beyond pricing, manufacturing remains one of the most significant challenges facing gene therapies. Most approved products are manufactured patient by patient. A single batch of CAR-T cells is made for one patient. A single lot of lentiviral-modified stem cells serves one patient. This individualized manufacturing model is fundamentally different from traditional pharmaceuticals, where millions of identical pills can be produced on a single production line.

Manufacturing failures, or "out-of-specification" results, mean that some patients who are enrolled and undergo preparative procedures do not receive a usable product. Vein-to-vein time (the period from cell collection to product infusion) for CAR-T therapies is typically three to five weeks, during which patients with aggressive cancers may deteriorate.

Companies are investing heavily in manufacturing improvements. Automated, closed-system manufacturing platforms are being developed to reduce variability and production time. Point-of-care manufacturing, where CAR-T cells are produced at or near the treatment center rather than shipped to a central facility, is being explored by several companies. And next-generation "off-the-shelf" allogeneic cell therapies, made from healthy donor cells rather than the patient's own cells, could eventually eliminate the need for individualized manufacturing altogether.

What Comes Next

The pipeline of gene therapies in late-stage clinical development suggests that the pace of approvals will continue to accelerate. Key areas of expansion include:

In vivo CRISPR therapies: Intellia Therapeutics is developing CRISPR therapies administered directly into the body, without the need for ex vivo cell manipulation. Their lead program for transthyretin amyloidosis (ATTR) has shown promising clinical data and could become the first in vivo CRISPR therapy to reach the market.
Base editing and prime editing: These next-generation editing technologies offer even greater precision than standard CRISPR-Cas9 and are advancing through clinical trials for conditions including sickle cell disease and hypercholesterolemia.
Additional CAR-T targets: New CAR-T therapies targeting antigens beyond CD19 and BCMA are in development for solid tumors, autoimmune diseases, and other hematologic malignancies.
Neurological diseases: Gene therapies for Huntington disease, amyotrophic lateral sclerosis (ALS), Alzheimer disease, and other neurological conditions are in various stages of clinical development.
Cardiovascular disease: Verve Therapeutics is pursuing gene editing to permanently lower LDL cholesterol, an approach that could eventually address the leading cause of death worldwide.

Conclusion

The list of FDA-approved gene therapies has grown from a single product in 2017 to more than 20 in early 2026. This expansion reflects not only scientific progress but also the maturation of regulatory frameworks, manufacturing processes, and payment models needed to bring these complex therapies to patients.

Each therapy on this list represents a potential cure or transformative treatment for patients who previously had limited or no options. For the child with SMA who can now walk, the adult with sickle cell disease who is free from pain crises, the boy with CALD who retains his cognitive function, and the cancer patient in long-term remission, gene therapy has already delivered on its promise.

The challenges that remain are real: high costs, manufacturing complexity, limited access, long-term safety monitoring, and the need to extend these benefits to patients around the world. But the trajectory is unmistakable. Gene therapy is no longer a future possibility. It is a present reality, and this list will only continue to grow.

Sources

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