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DelveInsight Evaluates a Robust Adeno-Associated Virus Vectors in Gene Therapy Pipeline as 70+ Influential Pharma Players to Set Foot in the Domain

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DelveInsight Evaluates a Robust Adeno-Associated Virus Vectors in Gene Therapy Pipeline as 70+ Influential Pharma Players to Set Foot in the Domain

March 15
19:09 2023
DelveInsight Evaluates a Robust Adeno-Associated Virus Vectors in Gene Therapy Pipeline as 70+ Influential Pharma Players to Set Foot in the Domain

DelveInsight’s, “Adeno Associated Virus Vectors in Gene Therapy Pipeline Insight 2023″ report provides comprehensive insights about 70+ companies and 235+ pipeline drugs in AAV vectors in gene therapy pipeline landscape. It covers the Adeno Associated Virus Vectors in Gene Therapy pipeline drug profiles, including Adeno Associated Virus Vectors in Gene Therapy clinical trial and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

For Adeno-Associated Virus Vectors in Gene Therapy Emerging drugs, the Adeno-Associated Virus Vectors in Gene Therapy pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The Adeno-Associated Virus Vectors in Gene Therapy pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.

 

In the Adeno-Associated Virus Vectors in Gene Therapy Pipeline Report, a detailed description of the drug is given which includes the mechanism of action of the drug, Adeno-Associated Virus Vectors in Gene Therapy clinical trials studies, Adeno-Associated Virus Vectors in Gene Therapy NDA approvals (if any), and product development activities comprising the technology, Adeno-Associated Virus Vectors in Gene Therapy collaborations, licensing, mergers and acquisition, funding, designations, and other product-related details.

 

Key takeaways from the Adeno-Associated Virus Vectors in Gene Therapy Pipeline Report

 

  • DelveInsight’s Adeno Associated Virus Vectors in Gene Therapy pipeline report depicts a robust space with 70+ active players working to develop 235+ pipeline therapies for Adeno Associated Virus Vectors in Gene Therapy treatment.

 

  • The leading Adeno Associated Virus Vectors in Gene Therapy Companies include Biomarin Pharmaceutical, Sarepta Therapeutics, Roche (Spark Therapeutics), Sangamo, Pfizer, NightstaRx, Freeline Therapeutics, Horama S.A, MeiraGTx, RegenxBio, Asklepios Biopharmaceutical, Audentes Therapeutics, and others.

 

  • Promising Adeno Associated Virus Vectors in Gene Therapy Pipeline Therapies include AAV – CNGB3, AAV2/5-RPGR, AAV OPTIRPE65, BMN 307, AAV RPE65, AGTC-402, and others.

 

  • The Adeno Associated Virus Vectors in Gene Therapy Companies and academics are working to assess challenges and seek opportunities that could influence AAV vectors in gene therapy R&D. The Adeno Associated Virus Vectors in Gene Therapy pipeline therapies under development are focused on novel approaches to treat/improve AAV vectors in gene therapy.

 

Request a sample and discover the recent breakthroughs happening in the Adeno-Associated Virus Vectors in Gene Therapy Pipeline landscape @ Adeno-Associated Virus Vectors in Gene Therapy Pipeline Outlook Report

 

Adeno-Associated Virus Vectors in Gene Therapy Overview

Out of the several viral vectors that have been used to date for delivering the genes of interest, the Adeno-associated viral (AAV) vector appears to be the safest and effective vehicle and can maintain long-term gene and protein expression following a single injection of the vector. AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases.

 

Recent Developmental Activities in the Adeno Associated Virus Vectors in Gene Therapy Treatment Landscape

 

  • In June 2021, BioMarin resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA).  In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase III GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.

 

  • AAV-RPGR is an investigational gene therapy for the treatment of patients with X-linked retinitis pigmentosa (XLRP) caused by disease-causing variants in the eye specific form of the RPGR gene (RPGR ORF15). AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function. MeiraGTx and development partner Janssen are currently conducting a Phase III clinical trial of AAV-RPGR in patients with XLRP with disease-causing variants in RPGR ORF15.

 

  • Timrepigene emparvovec is an AAV2 vector administered by subretinal injection, which aims to provide a functioning CHM gene and expression of the REP-1 protein to restore membrane trafficking and thereby slow, stop or potentially reverse decline in vision. Data from the Phase 1/2 studies demonstrated a slower rate of decline in visual acuity in patients treated with timrepigene emparvovec compared to untreated patients in the natural history study. In addition, some patients treated with timrepigene emparvovec showed improvements in visual acuity. The studies also demonstrated that timrepigene emparvovec was generally well tolerated with an acceptable safety profile. The safety and efficacy of a single subretinal injection of timrepigene emparvovec is currently being evaluated in the ongoing Phase III STAR study.

 

For further information, refer to the detailed Adeno-Associated Virus Vectors in Gene Therapy Drugs Launch, Adeno-Associated Virus Vectors in Gene Therapy Developmental Activities, and Adeno-Associated Virus Vectors in Gene Therapy News, click here for Adeno-Associated Virus Vectors in Gene Therapy Ongoing Clinical Trial Analysis

 

Adeno-Associated Virus Vectors in Gene Therapy Emerging Drugs Analysis

 

  • GS010: Gensight Biologics

LUMEVOQ® (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. “LUMEVOQ” was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

 

  • Valoctocogene roxaparvovec: BioMarin Pharmaceutical

Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A. It is currently in preregistration stage of development. In June 2021, BioMarin resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA).  In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase III GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.

 

  • DTX401: Ultragenyx Pharmaceutical

DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-α under control of the native promoter. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase-α activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. In a Phase I/II clinical study, all nine patients showed a clinical response, with significant reductions in the need for cornstarch and improvements in glucose control and other metabolic parameters compared to baseline. The drug is currently being evaluated in Phase III clinical trial to treat patients with Glycogen storage disease type I.

 

  • AAV5-RPGR: MeiraGTx

AAV-RPGR is an investigational gene therapy for the treatment of patients with X-linked retinitis pigmentosa (XLRP) caused by disease-causing variants in the eye specific form of the RPGR gene (RPGR ORF15). AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function. MeiraGTx and development partner Janssen are currently conducting a Phase III clinical trial of AAV-RPGR in patients with XLRP with disease-causing variants in RPGR ORF15.

 

  • Timrepigene emparvovec: Biogen

Timrepigene emparvovec is an AAV2 vector administered by subretinal injection, which aims to provide a functioning CHM gene and expression of the REP-1 protein to restore membrane trafficking and thereby slow, stop or potentially reverse decline in vision. Data from the Phase 1/2 studies demonstrated a slower rate of decline in visual acuity in patients treated with timrepigene emparvovec compared to untreated patients in the natural history study. In addition, some patients treated with timrepigene emparvovec showed improvements in visual acuity. The studies also demonstrated that timrepigene emparvovec was generally well tolerated with an acceptable safety profile. The safety and efficacy of a single subretinal injection of timrepigene emparvovec is currently being evaluated in the ongoing Phase III STAR study.

 

  • AMT 061: UniQure

AMT-061 (etranacogene dezaparvovec) is an experimental gene therapy that uniQure is developing to treat hemophilia B. AMT-061 uses a modified and harmless adeno-associated virus 5 (AAV5) to deliver a highly functional copy of the F9 gene, called FIX-Padua, to patients’ cells. The FIX-Padua gene version was shown to result in FIX clotting activity eight times greater than that associated with the standard F9 gene. As such, the one-time therapy — administrated directly into the bloodstream — is expected to increase FIX levels, helping to prevent and control bleeds. If approved, the therapy will be marketed globally by CSL Behring, which closed a commercialization and licensing agreement with uniQure in May 2021. UniQure and CSL Behring expect to file a regulatory application to the U.S. Food and Drug Administration (FDA) in early 2022 seeking AMT-061’s approval for hemophilia B. Research and Development.

 

  • RGX-314: REGENXBIO

RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina. REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye. According to the company’s pipeline, the drug is currently in the Phase III stage of development.

 

  • SPK-8011: Spark Therapeutics

Investigational SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter. The Food and Drug Administration (FDA) granted orphan-disease designation and breakthrough therapy designation in the US, while the European Commission has granted orphan designation to SPK-8011.

 

  • NFS-01: Neurophth

Investigational NR082, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized of NADH-dehydrogenase subunit 4 (ND4) gene under the control of the cytomegalovirus promoter and enhancer, is a novel ophthalmic injection that is being developed for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND4 mutations. It is currently being evaluated in Phase II/III clinical trial.

 

  • GT 005: Gyroscope Therapeutics

GT005 is designed as an AAV2-based one-time investigational gene therapy for GA secondary to AMD that is delivered under the retina. GT005 aims to restore balance to an overactive complement system, a part of the immune system, by increasing production of the CFI protein. Complement overactivation has been strongly correlated with the development and progression of AMD. The CFI protein regulates the activity of the complement system. It is believed that increasing CFI production could dampen the system’s overactivity and reduce inflammation, with the goal of preserving a person’s eyesight. Gyroscope is also evaluating GT005 in two Phase II clinical trials.

 

Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapeutics Assessment

There are approx. 70+ key companies which are developing the therapies for AAV vectors in gene therapy. The companies which have their AAV vectors in gene therapy drug candidates in the most advanced stage, i.e. Preregistration include, BioMarin Pharmaceutical.

 

Find out more about the Adeno-Associated Virus Vectors in Gene Therapy Pipeline Segmentation, Therapeutics Assessment, and Adeno-Associated Virus Vectors in Gene Therapy Emerging Drugs @ Adeno-Associated Virus Vectors in Gene Therapy Treatment Landscape

 

Scope of the Adeno Associated Virus Vectors In Gene Therapy Pipeline Report

 

  • Coverage- Global

 

  • Adeno Associated Virus Vectors in Gene Therapy Companies- Biomarin Pharmaceutical, Sarepta Therapeutics, Roche (Spark Therapeutics), Sangamo, Pfizer, NightstaRx, Freeline Therapeutics, Horama S.A, MeiraGTx, RegenxBio, Asklepios Biopharmaceutical, Audentes Therapeutics, and others.

 

  • Adeno Associated Virus Vectors in Gene Therapy Pipeline Therapies- AAV – CNGB3, AAV2/5-RPGR, AAV OPTIRPE65, BMN 307, AAV RPE65, AGTC-402, and others.

 

  • Adeno Associated Virus Vectors in Gene Therapy Segmentation: Product Type, Molecule Type, Mechanism of Action, Route of Administration

 

Dive deep into rich insights for drugs for Adeno-Associated Virus Vectors in Gene Therapy Pipeline Companies and Therapies, click here @ Adeno-Associated Virus Vectors in Gene Therapy Unmet Needs and Analyst Views

 

Table of Content

  1. Introduction
  2. Executive Summary
  3. Adeno-Associated Virus Vectors in Gene Therapy: Overview
  4. Adeno-Associated Virus Vectors in Gene Therapy Pipeline Therapeutics
  5. Adeno-Associated Virus Vectors in Gene Therapy Therapeutic Assessment
  6. Late Stage Products (Preregistration)
  7. Valoctocogene roxaparvovec: BioMarin Pharmaceutical
  8. Drug profiles in the detailed report…..
  9. Late Stage Products (Phase III)
  10. DTX401: Ultragenyx Pharmaceutical
  11. Drug profiles in the detailed report…..
  12. Mid Stage Products (Phase II)
  13. GT 005: Gyroscope Therapeutics
  14. Drug profiles in the detailed report…..
  15. Early stage products (Phase I/II)
  16. AXO-AAV-GM1: Sio Gene Therapies
  17. Drug profiles in the detailed report…..
  18. Early Stage Products (Phase I)
  19. HMI-203: Homology Medicine
  20. Drug profiles in the detailed report…..
  21. Preclinical stage products
  22. AXV101: Axovia Therapeutics
  23. Drug profiles in the detailed report…..
  24. Discovery stage products
  25. DINA-002: DiNAQOR
  26. Drug profiles in the detailed report…..
  27. Inactive Products
  28. Adeno-Associated Virus Vectors in Gene Therapy Key Companies
  29. Adeno-Associated Virus Vectors in Gene Therapy Key Products
  30. Adeno-Associated Virus Vectors in Gene Therapy- Unmet Needs
  31. Adeno-Associated Virus Vectors in Gene Therapy- Market Drivers and Barriers
  32. Adeno-Associated Virus Vectors in Gene Therapy- Future Perspectives and Conclusion
  33. Adeno-Associated Virus Vectors in Gene Therapy Analyst Views
  34. Adeno-Associated Virus Vectors in Gene Therapy Key Companies
  35. Appendix

 

Got Queries? Find out the related information on Adeno-Associated Virus Vectors in Gene Therapy Mergers and acquisitions, Adeno-Associated Virus Vectors in Gene Therapy Licensing Activities @ Adeno-Associated Virus Vectors in Gene Therapy Recent Trends, and Future Perspectives

 

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