A Comprehensive Analysis of 250+ Key Companies Strengthening Gene Therapy Clinical Trial Pipeline | DelveInsight

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Nov 02, 2022, 11:30 ET

Gene therapy clinical trial pipeline constitutes 250+ key companies continuously working towards developing 300+ gene therapies, analyzes DelveInsight

LAS VEGAS, Nov. 2, 2022 /PRNewswire/ -- DelveInsight's 'Gene Therapy Competitive Landscape – 2022' report provides comprehensive global coverage of available, marketed, and pipeline gene therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the gene therapy pipeline domain.

Key Takeaways from the Gene Therapy Pipeline Report

Over 250+ Gene Therapy companies are evaluating 300+ gene therapies in various stages of development, and their anticipated acceptance in the gene therapy market would significantly increase market revenue.
Leading gene therapy companies such as Novartis, Johnson & Johnson, Fibrocell Technologies, Pfizer, HELIXMITH Co., Ltd., Sarepta Therapeutics, Solid Biosciences Inc., Lexeo Therapeutics, Spark Therapeutics, Xalud Therapeutics, uniQure, Ultragenyx Pharmaceutical, Nanoscope Therapeutics, Homology Medicines, Freeline therapeutics, Amicus Therapeutics, REGENXBIO, Gyroscope Therapeutics, CSL, Sangamo Therapeutics, LYSOGENE, Asklepios BioPharmaceutical, Alcyone Therapeutics, Taysha Gene Therapies, Gensight Biologic, Vivet Therapeutics, BridgeBio, BioMarin Pharmaceutical, Neurophth Therapeutics, Abeona Therapeutics, Coave Therapeutics, Actus Therapeutics, Forge Biologics, Passage Bio, Aspa Therapeutics, Adrenas Therapeutics, ESTEVE, 4D Molecular Therapeutics, Bayer, LogicBio Therapeutics, Eli Lilly and Company, Adverum Biotechnologies, Libella Gene Therapeutics, Rocket Pharmaceuticals, Decibel therapeutics, DiNAQOR, Affinia Therapeutics, Lacerta Therapeutics, Atamyo Therapeutics, Apic Bio, Orchard Therapeutics, Nervosave Therapeutics, Maze Therapeutics, SOLA Biosciences, Benitec Biopharma, Hanugen Therapeutics, Exegenesis Bio, CANbridge Life Sciences Ltd, StrideBio, ENZERNA BIOSCIENCES INC., AAVogen, Capsigen, Code Biotherapeutics, Amarna Therapeutics, Poseida Therapeutics, Inc., bluebird bio, Genethon, Voyager Therapeutics, Jaguar Gene Therapy, LLC, Oxford Biomedica, Ocugen, Genprex, Beam Therapeutics, Neurogene Inc., Treeway, Oyster Point Pharma, Elicera Therapeutics AB, Generation Bio Co., Atsena Therapeutics, Precision Biosciences, Precigen, SELECTA BIOSCIENCES, Encoded Therapeutics, Graphite Bio, Nuevocor, M6P Therapeutics, SwanBio Therapeutics, AviadoBio, Myrtelle, Kriya Therapeutics, Inc., Senti Biosciences, Castle Creek Biosciences, Inc., Treadwell Therapeutics, Opus Genetics, Renovacor, and others are evaluating novel gene therapies candidate to improve the treatment landscape.
Key gene therapies in the pipeline in various stages of development include Zolgensma, Carvykti, Laviv, Provenge, Fordadistrogene movaparvovec, Engensis, SRP-9001, RGX-202, SGT-001, LX2006, SPK 3006, XT-150, AMT-161, UX810, MCO 010, HMI-102, FLT190, EtranaDez, FLT180a, GT005, RGX-121, AT GAA, Giroctocogene fitelparvovec, SPK-8011, LYS-SAF302, ACTUS-101, ACTX-401, TSHA 120, GS030, VTX-801, BBP-631, ADVM-022, AAV- hTERT, RP-A501, and others.
In October 2022, Astellas and Taysha Gene Therapies announced the strategic investment to support the development of Taysha's AAV-based gene therapy programs.
In October 2022, French companies formed a strategic alliance to cover cell and gene therapy (C>) GMP manufacturing.
In October 2022, AVROBIO announced the patient dosing completion in the first gene therapy clinical trial for cystinosis treatment.
In October 2022, Xcell Biosciences Inc. (Xcellbio) and aCGT Vector announced a collaboration today to improve manufacturing and analytic procedures used to develop personalized cell and gene therapies for cancer patients.
In August 2022, the FDA approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.
In March 2022, REGENXBIO announced that the FDA granted Rare Pediatric Disease Designation for RGX-202, which may entitle REGENXBIO to receive a priority review voucher which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred should a new BLA for RGX-202 be approved.
In January 2022, REGENXBIO Inc. announced the clearance of its Investigational New Drug application by the FDA to evaluate RGX-202, potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne) in a first-in-human clinical trial. RGX-202 is designed to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. RGX-202 uses REGENXBIO's proprietary NAV® AAV8 vector.
In November 2021, REGENXBIO Inc. announced that the US FDA granted Orphan Drug Designation for RGX-202, potential one-time gene therapy for the treatment of Duchenne muscular dystrophy.

Request a sample and discover the recent advances in gene therapy @Gene Therapy Competitive Landscape Report

Gene Therapy Overview

Gene therapy is the treatment of disease through the introduction of genetic material into cells. This review will look at gene transfer methods and current and potential applications for craniofacial regeneration, focusing on future development and design. Though non-viral gene delivery methods have a low gene transfer efficiency, they have the advantages of relative safety, low immunogenicity, ease of manufacture, and the lack of a DNA insert size limitation.

Find out more about gene therapy treatment @Gene Therapy Market

Gene Therapy Pipeline Analysis: Drug Profile

Fordadistrogene movaparvovec: Pfizer

Pfizer's Fordadistrogene movaparvovec is a recombinant adeno-associated virus serotype 9 (AAV9) capsid that contains a shortened version of the human dystrophin gene (mini-dystrophin) controlled by a human muscle-specific promotor. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism. The gene therapy demonstrated durable and statistically significant improvements in multiple efficacy endpoints measured 12 months after infusion in the Phase Ib study of PF-06939926 in ambulatory for boys with DMD.

LX2006: Lexeo Therapeutics

Lexeo Therapeutics drug, LX2006, is an AAV-based gene therapy candidate administered intravenously to treat Friedreich's ataxia cardiomyopathy, the leading cause of death in Friedreich's ataxia patients. LX2006 targets the cardiac manifestations of Friedreich's ataxia by delivering a functional frataxin gene, which promotes frataxin protein expression and restores mitochondrial function in myocardial cells. LX2006 reversed cardiac abnormalities in Friedreich's ataxia disease models in preclinical studies, improving cardiac function and survival while demonstrating a favorable safety profile. LX2006 has been granted Rare Pediatric Disease and Orphan Drug designation by the FDA for treating Friedreich's ataxia.

A snapshot of the Gene Therapy Pipeline Drugs mentioned in the report:

Gene Therapy Drugs	Company	Phase	Indication
Etranacogene dezaparvovec	CSL Behring/uniQure	Pre-registration	Hemophilia B
Engensis	Helixmith	Phase III	Diabetic foot ulcer; Diabetic neuropathies; Peripheral arterial disorders
Fordadistrogene movaparvovec	Pfizer	Phase III	Duchenne muscular dystrophy
RGX-121	REGENXBIO	Phase II/III	Mucopolysaccharidosis II
LYS-SAF302	LYSOGENE	Phase II/III	Mucopolysaccharidosis III
GT005	Gyroscope Therapeutics	Phase II	Dry age-related macular degeneration
SPK 3006	Spark Therapeutics	Phase II	Glycogen storage disease type II
FLT190	Freeline Therapeutics	Phase II	Fabry's disease
LX2006	Lexeo Therapeutics	Phase I/II	Cardiomyopathies

Learn more about the emerging gene therapies @Gene Therapy Clinical Trials

Scope of the Gene Therapy Competitive Landscape Report

Coverage: Global
Key Gene Therapy Companies: Novartis, Johnson & Johnson, Fibrocell Technologies, Pfizer, HELIXMITH Co., Ltd., Sarepta Therapeutics, Solid Biosciences Inc., Lexeo Therapeutics, Spark Therapeutics, Xalud Therapeutics, uniQure, Ultragenyx Pharmaceutical, Nanoscope Therapeutics, Homology Medicines, Freeline therapeutics, Amicus Therapeutics, REGENXBIO, Gyroscope Therapeutics, CSL, Sangamo Therapeutics, LYSOGENE, Asklepios BioPharmaceutical, Alcyone Therapeutics, Taysha Gene Therapies, Gensight Biologic, Vivet Therapeutics, BridgeBio, BioMarin Pharmaceutical, Neurophth Therapeutics, Abeona Therapeutics, Coave Therapeutics, Actus Therapeutics, Forge Biologics, Passage Bio, Aspa Therapeutics, Adrenas Therapeutics, ESTEVE, 4D Molecular Therapeutics, Bayer, LogicBio Therapeutics, Eli Lilly and Company, Adverum Biotechnologies, Libella Gene Therapeutics, Rocket Pharmaceuticals, Decibel therapeutics, DiNAQOR, Affinia Therapeutics, Lacerta Therapeutics, Atamyo Therapeutics, Apic Bio, Orchard Therapeutics, Nervosave Therapeutics, Maze Therapeutics, SOLA Biosciences, Benitec Biopharma, Hanugen Therapeutics, Exegenesis Bio, CANbridge Life Sciences Ltd, StrideBio, ENZERNA BIOSCIENCES INC., AAVogen, Capsigen, Code Biotherapeutics, Amarna Therapeutics, Poseida Therapeutics, Inc., bluebird bio, Genethon, Voyager Therapeutics, Jaguar Gene Therapy, LLC, Oxford Biomedica, Ocugen, Genprex, Beam Therapeutics, Neurogene Inc., Treeway, Oyster Point Pharma, Elicera Therapeutics AB, Generation Bio Co., Atsena Therapeutics, Precision Biosciences, Precigen, SELECTA BIOSCIENCES, Encoded Therapeutics, Graphite Bio, Nuevocor, M6P Therapeutics, SwanBio Therapeutics, AviadoBio, Myrtelle, Kriya Therapeutics, Inc., Senti Biosciences, Castle Creek Biosciences, Inc., Treadwell Therapeutics, Opus Genetics, Renovacor, and others
Key Gene Therapies in Pipeline: Zolgensma, Carvykti, Laviv, Provenge, Fordadistrogene movaparvovec, Engensis, SRP-9001, RGX-202, SGT-001, LX2006, SPK 3006, XT-150, AMT-161, UX810, MCO 010, HMI-102, FLT190, EtranaDez, FLT180a, GT005, RGX-121, AT GAA, Giroctocogene fitelparvovec, SPK-8011, LYS-SAF302, ACTUS-101, ACTX-401, TSHA 120, GS030, VTX-801, BBP-631, and others.

Dive deep into rich insights for approved gene therapies; visit @FDA-approved Gene Therapies

Table of Contents

1.	Gene Therapy Pipeline Report Introduction
2.	Gene Therapy Pipeline Report Executive Summary
3.	Gene Therapy Pipeline: Overview
4.	Analytical Perspective In-depth Commercial Assessment
5.	Gene Therapy Clinical Trial Therapeutics
6.	Gene Therapy Pipeline: Late Stage Products (Pre-registration)
7.	Gene Therapy Pipeline: Late Stage Products (Phase III)
8.	Gene Therapy Pipeline: Mid Stage Products (Phase II)
9.	Gene Therapy Pipeline: Early Stage Products (Phase I)
10.	Gene Therapy Pipeline Therapeutic Assessment
11.	Inactive Products in the Gene Therapy Pipeline
12.	Company-University Collaborations (Licensing/Partnering) Analysis
13.	Key Gene Therapy Companies
14.	Key Products in the Gene Therapy Pipeline
15.	Gene Therapy Unmet Needs
16.	Gene Therapy Market Drivers and Barriers
17.	Future Perspectives and Conclusion
18.	Analyst Views
19.	Appendix

For further information on gene therapy uses, reach out @Viral Vectors for Gene Therapy

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