The research conducted by Pharma orgs across the globe in the field of gene therapy has brought forth significant advancements and potential benefits for patients with various genetic disorders and diseases. These pharmaceutical companies have contributed to expanding our understanding of gene-based treatments, investigating the long-term safety and efficacy of experimental gene therapies, exploring real-world treatment patterns, developing innovative therapeutic options, and assessing the economic and quality of life outcomes associated with gene therapies. Their research endeavors pave the way for promising treatment options, precision interventions, improved patient outcomes, and potentially life-changing therapies for individuals affected by genetic conditions.
Below are just a few of the key advances this year. For access to Amplion’s complete coverage of the Gene Therapy space, contact us here.
Bristol-Myers Squibb has been the most active pharmaceutical company in gene therapy research so far this year, including the following key events:
1. A Study of CC-97540 in Participants With Severe, Refractory Systemic Lupus Erythematosus (SLE):
– Bristol Myers Squibb conducted a study to investigate the efficacy and safety of CC-97540 in participants with severe, refractory Systemic Lupus Erythematosus (SLE).
– The study aimed to assess the potential of CC-97540 as a therapeutic option for individuals with this challenging autoimmune condition.
2. Lisocabtagene Maraleucel as Second-Line Therapy for Large B-Cell Lymphoma: Primary Analysis of the Phase 3 TRANSFORM Study:
– Bristol Myers Squibb conducted a Phase 3 TRANSFORM study to evaluate the efficacy and safety of lisocabtagene maraleucel as second-line therapy for large B-cell lymphoma.
– The primary analysis of this study assessed the potential benefits of lisocabtagene maraleucel in treating patients with relapsed or refractory large B-cell lymphoma.
3. Phase 2 Results of Idecabtagene Vicleucel (Ide-Cel, Bb2121) in Japanese Patients with Relapsed and Refractory Multiple Myeloma:
– Bristol Myers Squibb reported Phase 2 results of idecabtagene vicleucel (Ide-Cel, Bb2121) in Japanese patients with relapsed and refractory multiple myeloma.
– The research aimed to evaluate the efficacy and safety of Ide-Cel in this patient population, providing insights into its potential as a treatment option.
4. Cost-Effectiveness of Idecabtagene Vicleucel Compared with Conventional Care in Triple-Class Exposed Relapsed/refractory Multiple Myeloma Patients in Canada and France:
– Bristol Myers Squibb conducted an analysis to assess the cost-effectiveness of idecabtagene vicleucel compared to conventional care in triple-class exposed relapsed/refractory multiple myeloma patients in Canada and France.
– The analysis aimed to provide economic insights into the value and feasibility of Ide-Cel as a therapeutic intervention for this specific patient population.
5. Use of a Real-World Synthetic Control Arm for Direct Comparison of Lisocabtagene Maraleucel and Conventional Therapy in Relapsed/refractory Large B-Cell Lymphoma:
– Bristol Myers Squibb explored the use of a real-world synthetic control arm to directly compare lisocabtagene maraleucel with conventional therapy in relapsed/refractory large B-cell lymphoma.
– The research aimed to provide comparative data on the efficacy and safety of different treatment approaches, assisting in treatment decision-making.
6. Ide-Cel or Standard Regimens in Relapsed and Refractory Multiple Myeloma:
– Bristol Myers Squibb investigated the use of idecabtagene vicleucel (Ide-Cel) compared to standard regimens in patients with relapsed and refractory multiple myeloma.
– The research aimed to evaluate the efficacy and safety of Ide-Cel in comparison to standard treatment options for this patient population.
These disclosures from Bristol Myers Squibb showcase their commitment to advancing gene therapy research and developing innovative treatment options for various diseases, including systemic lupus erythematosus, large B-cell lymphoma, and multiple myeloma. Their studies provide insights into the efficacy, safety, and cost-effectiveness of gene therapies and contribute to the scientific understanding of these conditions.
Janssen has been actively involved in various gene therapy research endeavors. Here is a summary of their notable research and clinical trial activity in 2023:
1. CAR- PRISM (PRecision Intervention Smoldering Myeloma):
– Janssen initiated the CAR-PRISM study, which focuses on precision intervention for smoldering myeloma.
– The study aims to explore the potential of CAR (Chimeric Antigen Receptor) therapies for treating smoldering myeloma, a precursor condition to multiple myeloma.
2. A Study of JNJ-90009530 in Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma (R/r B-NHL):
– Janssen conducted a study to assess the efficacy of JNJ-90009530 in patients with relapsed or refractory B-cell non-Hodgkin lymphoma (R/r B-NHL).
– The research aimed to evaluate the potential of this therapeutic candidate in providing benefits for individuals with this type of lymphoma.
3. A Study to Evaluate Intravitreal JNJ-81201887 (AAVCAGsCD59) Compared to Sham Procedure for the Treatment of Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD):
– Janssen initiated a study to evaluate the use of intravitreal JNJ-81201887 (AAVCAGsCD59) compared to a sham procedure for treating geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
– The study aimed to assess the potential of JNJ-81201887 in slowing down or preventing the progression of GA, a degenerative eye condition.
4. A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-Linked Retinitis Pigmentosa:
– Janssen conducted a study to evaluate the use of AAV5-hRKp.RPGR as a potential treatment for Japanese participants with X-linked retinitis pigmentosa.
– The research aimed to assess the safety and efficacy of this gene therapy in improving the visual outcomes for individuals with this inherited retinal disorder.
5. Ciltacabtagene Autoleucel, an Anti-B-Cell Maturation Antigen Chimeric Antigen Receptor T-Cell Therapy, for Relapsed/Refractory Multiple Myeloma: CARTITUDE-1 2-Year Follow-Up:
– Janssen provided a 2-year follow-up of the CARTITUDE-1 study, which evaluated the use of ciltacabtagene autoleucel, a Chimeric Antigen Receptor T-cell therapy, in relapsed or refractory multiple myeloma.
– The follow-up aimed to provide insights into the long-term efficacy and safety of ciltacabtagene autoleucel as a treatment option for relapsed or refractory multiple myeloma patients.
6. Efficacy and Safety of Cilta-Cel in Patients with Progressive Multiple Myeloma After Exposure to Other BCMA-Targeting Agents:
– Janssen investigated the efficacy and safety of cilta-cel, a BCMA (B-cell maturation antigen)-targeting CAR-T cell therapy, in patients with progressive multiple myeloma following exposure to other BCMA-targeting agents.
– The research aimed to assess the potential benefits of cilta-cel in patients who have not responded adequately to previous treatments.
These disclosures by Janssen highlight their commitment to advancing gene therapy research for conditions such as multiple myeloma, non-Hodgkin lymphoma, and retinal disorders. The studies provide insight into the potential benefits of CAR-T cell therapies and gene-based treatments, showcasing the company’s dedication to developing innovative therapeutic options.
GlaxoSmithKline made significant advancements in the field of gene therapy. Below is a summary of their notable developments:
1. Study of GSK3845097 in Previously Treated Participants With Advanced Synovial Sarcoma and Myxoid/Round Cell Liposarcoma:
– GlaxoSmithKline conducted a study to evaluate the efficacy and safety of GSK3845097 in participants with advanced synovial sarcoma and myxoid/round cell liposarcoma.
– The aim of the study was to investigate the potential of GSK3845097 as a treatment option for individuals who had previously received treatment for these specific types of cancer.
2. Study to Evaluate Safety and Antitumor Activity of Lete-Cel (GSK3377794) in HLA-A2+ Participants With NY-ESO-1 Positive Previously Untreated Advanced Synovial Sarcoma and Myxoid/Round Cell Liposarcoma:
– Another study conducted by GlaxoSmithKline focused on evaluating the safety and antitumor activity of Lete-Cel (GSK3377794) in participants with previously untreated advanced synovial sarcoma and myxoid/round cell liposarcoma.
– The study specifically targeted individuals who were HLA-A2+ and NY-ESO-1 positive, with the goal of assessing Lete-Cel’s potential as a treatment option for these specific patient populations.
3. Safety and Efficacy of Letetresgene Autoleucel Alone or with Pembrolizumab for Relapsed/refractory Multiple Myeloma:
– GlaxoSmithKline conducted a study to evaluate the safety and efficacy of Letetresgene Autoleucel, both as a standalone therapy and in combination with Pembrolizumab, for relapsed/refractory multiple myeloma.
– The study aimed to assess the treatment’s impact on patient outcomes and determine its potential as a therapeutic option for individuals with this particular type of cancer.
4. Using Bayesian Evidence Synthesis Methods to Incorporate Real-World Evidence in Surrogate Endpoint Evaluation:
– GlaxoSmithKline explored the use of Bayesian evidence synthesis methods in order to incorporate real-world evidence when evaluating surrogate endpoints.
– This approach enables researchers to leverage data from various sources to assess the suitability of surrogate endpoints in clinical trials, ultimately enhancing the evaluation process.
5. 2022 White Paper on Recent Issues in Bioanalysis:
– GlaxoSmithKline published a white paper in 2023 that addressed several recent issues in bioanalysis, specifically focusing on gene therapy, cell therapy, vaccines immunogenicity, and associated technologies.
– The paper covered topics such as FDA draft guidance on immunogenicity information in prescription drug labeling, the immunogenicity of LNP and viral vectors therapeutics/vaccines, prolongation effect, ADA affinity, risk-based approaches, and various assay techniques such as NGS, qPCR, and ddPCR.
6. Transfer of PRAME and NY-ESO Target Programs back to Adaptimmune:
– GlaxoSmithKline reached an agreement with Adaptimmune for the transfer of PRAME and NY-ESO target programs back to Adaptimmune.
– This transfer suggests a strategic collaboration between the two companies, potentially paving the way for further advancements in the development of therapies targeting these specific programs.
Amgen has made significant progress in gene therapy research. Here is a summary of their notable disclosures in this field:
1. Randomized, Double-Blind, Placebo-Controlled, Global Phase III Trial of Talimogene Laherparepvec Combined With Pembrolizumab for Advanced Melanoma:
– Amgen conducted a Phase III trial to evaluate the efficacy and safety of Talimogene Laherparepvec in combination with Pembrolizumab for advanced melanoma.
– The trial employed a randomized, double-blind, placebo-controlled design to assess the potential benefits of this combination therapy in patients with advanced melanoma.
2. Oncolytic Viral Kinetics Mechanistic Modeling of Talimogene Laherparepvec (T-VEC) a First-in-Class Oncolytic Viral Therapy in Patients with Advanced Melanoma:
– Amgen conducted mechanistic modeling of the oncolytic viral therapy Talimogene Laherparepvec (T-VEC) in patients with advanced melanoma.
– This research aimed to analyze the viral kinetics and understand the mechanisms of action of T-VEC, a first-in-class oncolytic viral therapy.
3. Talimogene Laherparepvec in Combination with Ipilimumab versus Ipilimumab Alone for Advanced Melanoma: 5-Year Final Analysis of a Multicenter, Randomized, Open-Label, Phase II Trial:
– Amgen performed a five-year final analysis of a Phase II trial evaluating Talimogene Laherparepvec in combination with Ipilimumab compared to Ipilimumab alone for advanced melanoma.
– The trial assessed the long-term efficacy and safety of the combination therapy compared to Ipilimumab alone in patients with advanced melanoma.
4. Final 5-Year Follow-Up Results Evaluating Neoadjuvant Talimogene Laherparepvec Plus Surgery in Advanced Melanoma: A Randomized Clinical Trial:
– Amgen conducted a randomized clinical trial with a five-year follow-up to evaluate the neoadjuvant use of Talimogene Laherparepvec combined with surgery in advanced melanoma.
– The trial aimed to assess the effects of this treatment approach on patient outcomes and determine its potential as a therapeutic strategy for advanced melanoma.
5. Clinical Pharmacology Profile of AMG 119, the First Chimeric Antigen Receptor T (CAR-T) Cell Therapy Targeting Delta-Like Ligand 3 (DLL3), in Patients with Relapsed/Refractory Small Cell Lung Cancer (SCLC):
– Amgen developed and studied AMG 119, the first Chimeric Antigen Receptor T (CAR-T) cell therapy targeting Delta-Like Ligand 3 (DLL3), in patients with relapsed/refractory Small Cell Lung Cancer (SCLC).
– The research focused on understanding the clinical pharmacology profile of AMG 119 and its potential as a therapeutic option for patients with relapsed/refractory SCLC.
6. A Closed, Autologous Bioprocess Optimized for TCR-T Cell Therapies:
– Amgen designed a closed, autologous bioprocess specifically optimized for TCR-T cell therapies.
– This development aimed to enhance the manufacturing process of TCR-T cell therapies, a promising treatment modality in cancer immunotherapy.
7. OncoVEX mGM-CSF Expands Tumor Antigen-Specific CD8+ T-Cell Response in Preclinical Models:
– Amgen conducted preclinical studies investigating OncoVEX mGM-CSF, a viral therapy, and its effects on expanding tumor antigen-specific CD8+ T-cell response.
– The research aimed to understand the potential of OncoVEX mGM-CSF in activating the immune system’s response against tumor antigens.
8. Quantitative Chromatographic Method Development for Residual Lidocaine in Topical Systems and Biological Samples:
– Amgen developed a quantitative chromatographic method for analyzing residual Lidocaine in topical systems and biological samples.
– This method aimed to provide an accurate and reliable means of assessing residual Lidocaine levels in both topical applications and biological matrices.
BioMarin has also made significant advances in gene therapy R&D – particularly for its size. Below are some of the key takeaways from their activities so far this year:
1. A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401):
– BioMarin conducted a long-term follow-up study to assess the outcomes of severe hemophilia A subjects who previously participated in a BioMarin clinical trial involving BMN 270, a gene therapy treatment.
– The research aimed to evaluate the long-term safety and efficacy of BMN 270 in providing sustained therapeutic benefits for individuals with severe hemophilia A.
2. Valoctocogene Roxaparvovec Gene Transfer in Participants with HIV:
– BioMarin initiated a study to evaluate the use of valoctocogene roxaparvovec, a gene therapy, in participants living with HIV.
– The research aimed to assess the safety and efficacy of valoctocogene roxaparvovec in this specific patient population.
3. Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A:
– BioMarin reported the two-year outcomes of valoctocogene roxaparvovec therapy for hemophilia A.
– The study aimed to provide insights into the long-term durability and efficacy of valoctocogene roxaparvovec as a gene therapy treatment for individuals with hemophilia A.
4. Matching-Adjusted Indirect Comparison of Bleeding Outcomes in Severe Haemophilia A:
– BioMarin conducted a matching-adjusted indirect comparison to evaluate bleeding outcomes in severe hemophilia A patients treated with valoctocogene roxaparvovec gene therapy, emicizumab prophylaxis, or factor VIII replacement prophylaxis.
– The research aimed to compare the efficacy of different treatment approaches and provide insights into the clinical outcomes of valoctocogene roxaparvovec.
5. Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A:
– BioMarin evaluated the health-related quality of life in individuals with severe hemophilia A following valoctocogene roxaparvovec gene therapy.
– The study aimed to assess the impact of gene therapy on patients’ overall well-being and functional outcomes.
These disclosures by BioMarin reflect their dedication to advancing gene therapy research for the treatment of severe hemophilia A. The studies demonstrate their commitment to evaluating long-term safety, efficacy, and quality of life outcomes in individuals receiving gene therapies. They provide valuable insights into the potential of gene therapies like BMN 270 and valoctocogene roxaparvovec to transform the lives of patients with severe hemophilia A.
Pfizer has been actively involved in gene therapy research. Here is a summary of their noteworthy activity in 2023:
1. A Study to Understand the Long-Term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy:
– Pfizer conducted a study to investigate the long-term safety and effects of an experimental gene therapy for Duchenne Muscular Dystrophy.
– The aim of the study was to understand the potential of this gene therapy in providing long-term benefits and improving the outcomes for individuals with this debilitating genetic disorder.
2. Real-World Treatment Patterns of Novel Drugs in Relapsed or Refractory Acute Lymphoblastic Leukemia Patients in Japan:
– Pfizer performed a real-world analysis to assess the treatment patterns of novel drugs in patients with relapsed or refractory Acute Lymphoblastic Leukemia (ALL) in Japan.
– The research aimed to gain insights into the real-world usage and effectiveness of these novel drugs, ultimately enhancing treatment strategies for ALL patients.
3. Development of an icIEF Assay for Monitoring AAV Capsid Proteins and Application to Gene Therapy Products:
– Pfizer focused on developing an icIEF (capillary isoelectric focusing) assay for monitoring AAV (adeno-associated virus) capsid proteins in gene therapy products.
– This assay development aimed to provide a robust method for evaluating the quality and characteristics of gene therapy products that employ AAV vectors.
4. Comparing Molecular and Computational Approaches for Detecting Viral Integration of AAV Gene Therapy Constructs:
– Pfizer conducted research comparing molecular and computational approaches for detecting viral integration of AAV gene therapy constructs.
– The study aimed to explore different methods for analyzing and assessing the integration of AAV vectors into the host genome, providing insights into the safety and efficacy of AAV-based gene therapies.
5. Lentiviral Gene Therapy Reverts GPIX Expression and Phenotype in Bernard-Soulier Syndrome Type C:
– Pfizer investigated the use of lentiviral gene therapy to reverse GPIX expression and correct the phenotype in individuals with Bernard-Soulier Syndrome Type C.
– The research aimed to evaluate the potential of lentiviral gene therapy as a treatment option for this rare bleeding disorder caused by a genetic mutation.
6. FDA Accepts Pfizer’s Application for Hemophilia B Gene Therapy Fidanacogene Elaparvovec:
– Pfizer successfully submitted an application to the FDA for their gene therapy product, fidanacogene elaparvovec, intended for the treatment of Hemophilia B.
– The acceptance of the application indicates a significant step forward in the development and potential approval of this gene therapy for individuals with Hemophilia B.
These disclosures by Pfizer reflect their commitment to advancing gene therapy and developing innovative treatments for various genetic disorders and diseases. The research covers a broad range of areas, including long-term safety assessments, real-world treatment patterns, assay development, computational analysis, and the development of potential gene therapy products.
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