Gene Therapy Competitive Landscape Report 2025 | DelveInsight

DelveInsight's, "Gene Therapy Competitive landscape, 2025," report provides comprehensive insights about 180+ companies and 200+ drugs in Gene Therapy Competitive landscape. It covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Gene Therapy Competitive Landscape Report

* On 17 August 2025, MeiraGTx LLC conducted a study of individual participation in this study will be approximately 54 months for participants who received AAV-GAD in Study MGT-GAD-025 and approximately 60 months for participants who received sham surgery in Study MGT-GAD-025.
* On 15 August 2025, Janssen Research & Development LLC organized a Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene.
* On 13 August 2025, REGENXBIO Inc . announced a phase I/II/III study to evaluate the safety, tolerability, pharmacodynamics (microdystrophin protein levels), pharmacokinetic, and clinical efficacy of RGX-202 when administered IV as one-time dose to ambulant male participants with Duchenne. A comprehensive, short-term, prophylactic immunosuppression regimen will be administered during treatment to mitigate a potential immune response. This study is being conducted in three sequential parts: a phase I/II study (Part 1), a phase 3 pivotal study (Part 2) and a confirmatory study (Part 3). Part 1 will study a one-time dose of RGX-202 (1x10^14 or 2x10^14 GC/kg) in up to 15 participants with Duchenne.
* DelveInsight's Gene Therapy Competitive landscape report depicts a robust space with 180-+ active players working to develop 200+ pipeline therapies for Gene Therapy treatment.
* The leading Gene Therapy Companies such as Pfizer, Ultragenyx Pharmaceutical Inc, Rocket Pharmaceuticals, Adverum Biotechnologies, Kyverna Therapeutics, Vivet Therapeutics, HELIXMITH Co, Rocket Pharmaceuticals, Bristol-Myers Squibb, Wellington Zhaotai Therapies, Reyon Pharmaceutical, Neowise Biotechnology, Orna Therapeutics, Genflow Biosciences, Xylocor Therapeutics, Kolon TissueGene, Allogene Therapeutics, Nanjing IASO Biotherapeutics, Shanghai Vitalgen BioPharma, Rui Therapeutics, Obsidian Therapeutics, YolTech Therapeutics, Neurotech USA, Beacon Therapeutics, REGENXBIO and others.
* Promising Gene Therapy Therapies such as Tipifarnib, Sucrose (3%), Chemotherapy, UX111, SPK-8011, SBT101, Abemaciclib, PI3K Inhibitor paxalisib, Entrectinib and others.

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Gene Therapy Marketed Therapies

* Novartis

Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, company use innovative science and digital technologies to create transformative treatments in areas of great medical need. In quest to find new medicines, consistently rank among the world's top companies investing in research and development. Novartis products reach more than 750 million people globally and are finding innovative ways to expand access to company latest treatments. Novartis Gene Therapies is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, company is working to turn promising gene therapies into proven treatments.

* Zolgensma

Zolgensma (onasemnogene abeparvovec) is the only gene therapy for spinal muscular atrophy (SMA) and the only SMA treatment designed to directly address the genetic root cause of the disease by replacing the function of the missing or non-working SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. Zolgensma was approved by the US Food and Drug Administration for the treatment of pediatric patients less than 2years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. . Zolgensma is now approved in more than 40 countries and more than 2,000 patients have been treated with Zolgensma globally across clinical trials, managed access programs, and in the commercial setting.

* Spark Therapeutics

Spark Therapeutics, is a fully integrated company committed to discovering, developing and delivering gene therapies, challenge the inevitability of genetic diseases, including blindness, hemophilia and neurodegenerative diseases. The company have successfully applied our technology in the first FDA-approved gene therapy in the U.S. for a genetic disease, and currently have three programs in clinical trials, including product candidates that have shown promising early results in patients with hemophilia. At Spark, there is the path to a world where no life is limited by genetic disease.

* LUXTURNA

LUXTURNA is the first FDA-approved gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease (IRD) and the first adeno-associated virus (AAV) vector gene therapy approved in the U.S. LUXTURNA was approved by FDA under Priority Review and previously received orphan drug and breakthrough therapy designations from FDA. With the approval of LUXTURNA, FDA will issue to Spark Therapeutics a Rare Pediatric Disease Priority Review Voucher for a Priority Review of a subsequent marketing application for a different product. Spark Therapeutics' Marketing Authorization Application (MAA) for LUXTURNA is currently under review with the European Medicines Agency (EMA). LUXTURNA also has received orphan product designations from EMA.

Gene Therapy Pipeline Therapies

* Pfizer

Pfizer Inc. is a global biopharmaceutical company with a rich history dating back to 1849 when it was founded by Charles Pfizer and Charles Erhart in Brooklyn, New York. The company is engaged in the discovery, development, manufacture, and sale of medicines and vaccines across various therapeutic areas such as vaccines, oncology, internal medicine, hospital, inflammation and immunology, and rare diseases. Pfizer's dedication to global health is evident through its extensive portfolio of medicines and vaccines that aim to enhance wellness, prevention, treatments, and cures for various diseases. The company collaborates with healthcare providers, governments, and communities worldwide to expand access to reliable and affordable healthcare services.

* Fordadistrogene movaparvovec

Fordadistrogene movaparvovec is an investigational recombinant adeno-associated virus serotype 9 (AAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The AAV9 capsid was chosen as the delivery mechanism because of its potential to target muscle tissue. The FDA has lifted a clinical hold on Pfizer's phase III clinical trial of its investigational gene therapy, fordadistrogene movaparvovec, in patients with Duchenne muscular dystrophy (DMD). Regulatory and ethics approvals to resume the CIFFREO Phase III ambulatory study, including the FDA's lift of its clinical hold, follow reviews of data and protocol amendments. Currently the drug is in Phase III stage of development for the treatment of Duchenne muscular dystrophy

* Ultragenyx Pharmaceutical Inc

Kyowa Kirin strives to create and deliver novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company with a more than 70-year heritage, the company apply cutting-edge science including expertise in antibody research and engineering, to address the needs of patients and society across multiple therapeutic areas including Nephrology, Oncology, Immunology/Allergy and Neurology. Across our four regions - Japan, Asia Pacific, North America and EMEA/International - The company focus on purpose, to make people smile, and are united by our shared values of commitment to life, teamwork/Wa, innovation, and integrity.

* DTX401

DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase- using a single intravenous infusion. Long term Phase I/II data demonstrate an acceptable safety profile and durability of response. All treated patients have experienced stable or improved glucose control while tapering or discontinuing oral glucose replacement therapy with cornstarch after receiving DTX401. The Phase III GlucoGene study is underway to evaluate the ability of DTX401 to reduce the use of cornstarch while maintaining or improving glucose control as well as the therapy's impact on patients' quality of life. DTX401 was granted Orphan Drug Designation in the United States, EU and United Kingdom, and Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation in the United States. DTX401 was accepted into the EMA's Priority Medicines program (PRIME), enabling more frequent interactions with the EMA and the potential for an accelerated approval.The drug has recently completed Phase III clinical trial for the treatment of patients with Glycogen storage disease type I.

* Rocket Pharmaceuticals

Rocket Pharmaceuticals, Inc. is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rocket's multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's lead clinical program is a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. Preclinical studies of additional bone marrow-derived disorders are ongoing and target Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO). Rocket's first AAV-based gene therapy program targets Danon disease, a rare neuromuscular and cardiovascular disease.

* RP-L102

RP-L102 is a gene therapy product containing autologous (patient-derived) hematopoietic stem cells (HSCs) that have been genetically modified with a lentiviral vector to contain a functional copy of the FANCA gene. If given early in life, RP-L102 gene therapy has the potential to serve as a preventative measure, correcting bone marrow cells before there is progression to severe bone marrow failure. RP-L102 has been granted Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations for the treatment of Fanconi Anemia type A in the United States and Orphan Drug designation in Europe. The drug is currently in Phase II stage of development for the treatment of patients with Fanconi Anemia (FA).

* Adverum Biotechnologies

Adverum Biotechnologies is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with neovascular or wet age-related macular degeneration.

* ADVM-022

ADVM022 is being developed for the treatment of wet AMD. ADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. Unlike other ophthalmic gene therapies that require a surgery to administer the gene therapy under the retina (sub-retinal approach) ADVM-022 has the advantage of being administered as a one-time IVT injection in the office and is designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance, and improve vision outcomes for patients with wet AMD. The OPTIC trial is designed as a multi-center, open-label, dose-ranging, safety and efficacy trial of ADVM-022 in patients with wet AMD who have demonstrated responsiveness to anti-VEGF treatment. Patients in OPTIC are treatment experienced, and previously required frequent anti-VEGF injections to manage their wet AMD and to maintain functional vision. The drug is currently in Phase II stage of development for the treatment of patients with Wet AMD.

* Kyverna Therapeutics

Kyverna is a patient-centered, clinical-stage biopharmaceutical company focused on developing cell therapies for patients suffering from autoimmune diseases. KYV-101 is advancing through clinical development across two broad areas of autoimmune disease: rheumatology and neurology, including two ongoing multi-center, open-label Phase I trials of KYV-101 in the United States and Germany for patients with lupus nephritis. Kyverna's pipeline includes next-generation chimeric antigen receptor (CAR) T-cell therapies in both autologous and allogeneic formats with properties intended to be well suited for use in B cell-driven autoimmune diseases. By advancing more than one mechanism for taming autoimmunity, Kyverna is positioned to act on its mission of transforming how autoimmune diseases are treated.

* KYV-101

KYV-101 is an autologous, fully human CD19 CAR T-cell product candidate for use in B cell-driven autoimmune diseases. The CAR in KYV-101 was designed by the National Institutes of Health (NIH) to improve tolerability and tested in a 20-patient Phase I trial in oncology. Results were published by the NIH in Nature Medicine. Kyverna is currently conducting trials of KYV-101 in patients with lupus nephritis, an autoimmune disease in which more than half of patients do not achieve a complete response to current therapies and are at risk of developing kidney failure. Additional clinical trials of KYV-101 in systemic sclerosis, and myasthenia gravis are in preparation. The drug is currently in Phase I/II stage of development for the treatment of patients with Lupus nephritis.

* Vivet Therapeutics

Vivet Therapeutics is a private, clinical-stage biotech company developing novel and long-lasting gene therapies for rare inherited metabolic conditions, including Wilson's disease. Vivet's gene therapy platform uses recombinant adeno-associated viruses (rAAVs) as vectors and has initiated two clinical programs and four pre-clinical assets to date. Its most advanced therapy is VTX-801, a novel gene therapy for Wilson's disease, with key clinical read-outs expected by the end of 2024. Vivet Therapeutics was founded in 2016 by CEO Dr Jean-Philippe Combal and CSO Dr Gloria Gonzalez-Aseguinolaza and is led by a highly experienced management team with deep expertise developing gene therapies and orphan drugs.

* VTX-802

VTX-802 is an AAV-based gene therapy for patients with Progressive Familial Intrahepatic Cholestasis Type 2. It delivers a codon-optimized version of the BSEP gene to the liver and is currently under preclinical testing. The drug is currently in Preclinical stage of development for the treatment of patients with Intrahepatic cholestasis.

Gene Therapy Analytical Perspective by DelveInsight

* In-depth Commercial Assessment: Gene Therapy Collaboration Analysis by Companies

The Report provides in-depth commercial assessment of drugs that have been included, which comprises collaboration, agreement, licensing and acquisition - deals values trends. The sub-segmentation is described in the report which provide company-company collaboration (licensing/partnering), company academic collaboration and acquisition analysis in tabulated form.

* Gene Therapy Competitive Landscape

The report comprises of comparative assessment of Companies (by therapy, development stage, and technology).

Gene Therapy Report Assessment

* Company Analysis
* Therapeutic Assessment
* Pipeline Assessment
* Inactive drugs assessment
* Unmet Needs

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Scope of the Gene Therapy Competitive Landscape Report

* Coverage- Global
* Gene Therapy Companies- Pfizer, Ultragenyx Pharmaceutical Inc, Rocket Pharmaceuticals, Adverum Biotechnologies, Kyverna Therapeutics, Vivet Therapeutics, HELIXMITH Co, Rocket Pharmaceuticals, Bristol-Myers Squibb, Wellington Zhaotai Therapies, Reyon Pharmaceutical, Neowise Biotechnology, Orna Therapeutics, Genflow Biosciences, Xylocor Therapeutics, Kolon TissueGene, Allogene Therapeutics, Nanjing IASO Biotherapeutics, Shanghai Vitalgen BioPharma, Rui Therapeutics, Obsidian Therapeutics, YolTech Therapeutics, Neurotech USA, Beacon Therapeutics, REGENXBIO and others.
* Gene Therapy Analytical Perspective: In-depth Commercial Assessment
* Gene Therapy Unmet Needs, Preclinical and Discovery Stage Products

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Table of Contents

* Introduction
* Executive Summary
* Gene Therapy: Overview
* Gene Therapy -Analytical Perspective: In-depth Commercial Assessment
* Competitive Landscape
* Therapeutic Assessment
* Gene Therapy: Company and Product Profiles (Marketed Therapies)
* Novartis
* Zolgensma
* Gene Therapy: Company and Product Profiles (Pipeline Therapies)
* Late Stage Products (Phase III)
* Pfizer
* Fordadistrogene movaparvovec
* Mid Stage Products (Phase II)
* Rocket Pharmaceuticals
* RP-L102
* Early Stage Products (Phase I/II)
* Kyverna Therapeutics
* KYV-101
* Preclinical and Discovery Stage Products
* Vivet Therapeutics
* VTX-802
* Inactive Products
* Gene Therapy- Unmet needs
* Gene Therapy - Market drivers and barriers
* Appendix

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