Gene Therapy Clinical Trial Pipeline Gains Momentum: 180+ Companies Lead the Charge in Pioneering New Treatments | DelveInsight

New York, USA, Oct. 22, 2024 (GLOBE NEWSWIRE) — Gene Therapy Clinical Trial Pipeline Gains Momentum: 180+ Companies Lead the Charge in Pioneering New Treatments | DelveInsight

The gene therapies market is experiencing significant growth, driven by advancements in research and technology. According to DelveInsight, the gene therapy clinical trial pipeline features over 180 key companies actively engaged in the development of more than 200 gene therapies. This dynamic landscape highlights the increasing investment and innovation in the sector, promising new treatment options for various diseases.

DelveInsight’s ‘Gene Therapy Competitive Landscape – 2024‘ report provides comprehensive global coverage of available, marketed, and pipeline gene therapies in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space and future growth potential of the gene therapy competitive domain.

Key Takeaways from the Gene Therapy Pipeline Report

• Over 180+ companies are evaluating 200+ 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 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 are evaluating novel gene therapy candidates to improve the treatment landscape.
• Key gene therapies in the pipeline in various stages of development include DTX401, RP-L102, ADVM-022, KYV-101, VTX-802, RP A501, CD19-NEX-T, GP28 zT2, RY 105, WZTL 002, NW 301V, Research programme: in-vivo CRISPR editing gene therapies, Research programme: sarcopenia therapeutics, Encoberminogene rezmadenovec, Tonogenchoncel-L, ALLO 605, Equecabtagene autoleucel, VGN R09, KN 5501, OBX 115, YOLT 201, Revakinagene taroretcel, Laruparetigene zosaparvovec, RGX 121, and others.

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

Gene Therapy Overview

Gene therapy is a groundbreaking medical approach that aims to treat or prevent diseases by directly modifying the genes within an individual’s cells. By delivering therapeutic genes into a patient’s cells, this technique seeks to address the underlying genetic defects that contribute to various diseases, particularly genetic disorders, cancers, and certain viral infections. The fundamental principle of gene therapy involves replacing faulty genes, inactivating or knocking out disease-causing genes, or introducing new genes to help fight a disease. This innovative treatment holds the potential to transform the management of previously incurable conditions, offering hope to patients with rare genetic disorders or advanced diseases.

One of the most promising aspects of gene therapy is its ability to provide long-lasting effects with potentially fewer side effects compared to traditional treatments like chemotherapy or long-term use of medications. Advances in technologies such as CRISPR/Cas9 and viral vectors have significantly improved the precision and efficiency of gene delivery systems. These methods enable researchers and clinicians to target specific genes with greater accuracy, reducing the risk of unintended genetic changes. Clinical trials have shown encouraging results, with some gene therapies successfully curing conditions such as spinal muscular atrophy and certain types of inherited blindness.

However, gene therapy is not without challenges and ethical considerations. Issues such as the potential for immune reactions to the viral vectors used for gene delivery, the long-term effects of gene modification, and concerns regarding germline editing raise important questions about the safety and regulation of these therapies. Additionally, the high cost of development and treatment presents barriers to accessibility for many patients. As research continues and the field evolves, it will be crucial to balance the promise of gene therapy with thorough ethical oversight and equitable access to ensure that its benefits can be realized by all patients in need.

Gene Therapy Pipeline Analysis: Drug Profile

ZOLGENSMA: Novartis

ZOLGENSMA (onasemnogene abeparvovec) is the sole gene therapy for spinal muscular atrophy and the only treatment specifically designed to target the genetic cause of the disease. It works by replacing the function of the missing or defective SMN gene, effectively halting disease progression through sustained expression of SMN protein after a single intravenous infusion. The FDA approved ZOLGENSMA for pediatric patients under the age of 2 who have spinal muscular atrophy due to bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Currently, ZOLGENSMA has received approval in over 40 countries, with more than 2,000 patients treated worldwide through clinical trials, managed access programs, and commercial use.

LUXTURNA: Spark Therapeutics

LUXTURNA is the first gene therapy approved by the FDA for a genetic condition, making it the only pharmacological treatment available for inherited retinal diseases. It is also the first AAV vector gene therapy approved in the US. The FDA granted LUXTURNA Priority Review status and it has previously been designated as an orphan drug and breakthrough therapy. Following this approval, the FDA will provide Spark Therapeutics with a Rare Pediatric Disease Priority Review Voucher to expedite the review of a future marketing application for another product. Currently, Spark Therapeutics’ MAA for LUXTURNA is under review by the EMA, which has also granted it orphan product designations.

Find out more about FDA-approved gene therapies @ Gene Therapy Analysis

Gene Therapy Market Dynamics

The gene therapy market is witnessing significant growth, driven by advancements in technology, increasing investments, and a growing understanding of genetic diseases. As researchers unlock the potential of gene editing tools like CRISPR, ZFNs, and TALENs, the landscape of gene therapy is evolving rapidly. These technologies enable precise modifications at the genetic level, paving the way for innovative treatments for a range of conditions, from rare genetic disorders to more common diseases like cancer. The rise in research activities and clinical trials highlights the expanding capabilities of gene therapy, leading to an increase in approved therapies and a promising pipeline of candidates.

Market dynamics are also influenced by regulatory frameworks and reimbursement policies. Authorities like the FDA and EMA are streamlining the approval process for gene therapies, recognizing their potential to address unmet medical needs. However, the complexity of these therapies presents challenges in terms of regulatory oversight and safety assessments. Additionally, the high costs associated with gene therapies have raised concerns about accessibility and reimbursement. Market players are collaborating with governments and insurance companies to establish pricing models that reflect the long-term value of these treatments, thus promoting wider adoption.

Furthermore, the competitive landscape is intensifying as established pharmaceutical companies and biotech startups alike seek to capitalize on the potential of gene therapy. Collaborations, mergers, and acquisitions are becoming increasingly common as companies aim to bolster their research and development capabilities and expand their product portfolios. Strategic partnerships with academic institutions and research organizations are also prevalent, as they provide access to cutting-edge research and innovations. This dynamic environment fosters innovation, enabling faster development and commercialization of new gene therapies.

Finally, public perception and awareness of gene therapy play a critical role in shaping market dynamics. As success stories emerge and media coverage increases, patient and healthcare provider awareness of the potential benefits of gene therapy grows. However, ethical considerations surrounding gene editing, especially in germline modifications, pose challenges that could impact public acceptance and regulatory policies. Educating stakeholders and fostering open dialogues about the benefits and risks of gene therapy will be essential for the market’s sustained growth and acceptance in the healthcare ecosystem.

To know more about gene therapies, visit @ Gene Therapy Market Insights 

A snapshot of the pipeline gene therapies mentioned in the report:

Discover more about gene therapies in clinical development @ Gene Therapy in Clinical Trials

Key Developments in the Gene Therapy Domain

• In July 2024, Pfizer discontinued the phase III CIFFREO trial evaluating investigational gene therapy fordadistrogene movaparvovec. Earlier the gene therapy also did not achieve the study’s main goal of enhancing motor function in boys aged 4 to 7 years compared to a placebo.
• In July 2024, Miltenyi Biotec announced the signing of a Letter of Intent with the Translational Health Science and Technology Institute (THSTI). With this partnership, both organizations aim to address the growing need for innovative treatments in the fight against cancer by developing innovative cell and gene therapies.
• In May 2024, Taysha Gene Therapies received a regenerative medicine advanced therapy (RMAT) designation for its gene therapy candidate TSHA-102 from the US Food and Drug Administration (FDA) to treat Rett syndrome.
• In April 2024, Charles River Laboratories International, Inc. announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration with Axovia Therapeutics Ltd. Charles River will manufacture High Quality (HQ) gene of interest plasmid to support the development of Axovia’s gene therapies for ciliopathies, including Bardet-Biedl Syndrome (BBS), a condition with limited treatment options and no cure.
• In April 2024, Ginkgo Bioworks successfully completed the gene therapy collaboration with Biogen announced in May 2021. The companies aimed to advance the industry standard for manufacturing recombinant adeno-associated virus (AAV)-based vectors. The collaboration achieved its goals of enhancing the AAV production titers of Biogen’s gene therapy manufacturing processes.
• In March 2024, Seattle Children’s Research Institute and Genezen unveiled a strategic manufacturing partnership for Seattle Children’s Research Institute’s X-linked agammaglobulinemia (XLA) program. This collaboration is focused on leveraging Genezen’s viral vector process development and cGMP manufacturing expertise to advance Seattle Children’s XLA gene therapy program.
• In March 2024, Taysha Gene Therapies announced that they had received the needed approval to move forward with dose escalation in its Phase I/II trial testing TSHA-102, its gene therapy candidate for Rett syndrome, in adolescents and adults.
• In February 2024, the FDA approved the first cell therapy for a solid tumor, MIP Discovery secured £7 million in Series A financing to drive the commercialization of novel synthetic affinity reagents within the cell and gene therapy space, and Charles River Laboratories International has expanded its portfolio of human pluripotent stem cells through a collaboration with Pluristyx.

Scope of the Gene Therapy Competitive Landscape Report 

• Coverage: Global
• Key Gene Therapy Companies: 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
• Key Gene Therapies in Pipeline: DTX401, RP-L102, ADVM-022, KYV-101, VTX-802, RP A501, CD19-NEX-T, GP28 zT2, RY 105, WZTL 002, NW 301V, Research programme: in-vivo CRISPR editing gene therapies, Research programme: sarcopenia therapeutics, Encoberminogene rezmadenovec, Tonogenchoncel-L, ALLO 605, Equecabtagene autoleucel, VGN R09, KN 5501, OBX 115, YOLT 201, Revakinagene taroretcel, Laruparetigene zosaparvovec, RGX 121, and others.

Table of Contents

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

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