Category Archives: Gene therapy

Jessica Merryfield

Naomi Kautz

Advancing Gene Therapy Development: Key Regulatory and Clinical Trial Considerations

By Naomi Kautz, Vice President, Regulatory Affairs, Premier Consulting

By Jessica Merryfield, Senior Director, Program Delivery, Rare Disease, Premier Research

Insights into the human genome have created myriad therapeutic opportunities for previously untreatable diseases. Gene therapy offers promise for addressing unmet medical needs across therapeutic areas and even the potential for curing certain genetically based conditions. As research tools and technology become more sophisticated, gene therapy development is accelerating at an unprecedented pace. According to the American Society of Gene & Cell Therapy, there are currently 19 gene therapies, including genetically modified cell therapies, approved for clinical use globally.[1] Nearly 1,900 gene therapies are in development, of which 34 are in Phase 3 or pre-registration trials.[1]

Gene therapy development is a complex endeavor, with evolving regulations and complicated study logistics. In this article, we explore the regulatory and clinical trial landscape for gene therapy trials and offer strategies for successfully negotiating the challenges of executing these studies.

Regulatory Landscape

U.S. Framework

Due to their unique mechanism of action and the associated novel risks, gene therapy products are subject to some regulations that are not applied to small molecule therapeutics. In recent years, coinciding with research advances and marketing approvals, the gene therapy regulatory landscape has shifted from high-level guidance to more detailed guidelines focused on testing, manufacturing, or specific disease states. Since January 2020, the U.S. Food and Drug Administration (FDA) has issued ten guidance documents for gene therapy program sponsors to align with the advances in research.[2]

A key feature of the FDAs recent guidance documents on gene therapy is an emphasis on weighing potential risks identified in nonclinical data when designing clinical trials. Such risks include genomic integration of the gene therapy product, genome editing, prolonged transgene expression, latency, and persistent infections. Additionally, biodistribution studies have become increasingly relevant in preclinical gene therapy studies. In addition, the National Institutes of Health recently released guidelines on biosafety practices and containment strategies for constructing and handling gene therapy products.[3]

EU Framework

In the European Union (EU), the overarching gene therapy guideline is the European Medicines Agency (EMA) Guideline on the quality, non-clinical, and clinical aspects of gene therapy medicinal products. Each member state, however, has its own directives that may require additional reviews by a relevant health authority, ethics committee (EC), or genetically modified organism (GMO) authority. To ease the complexity, the EMA created the Committee for Advanced Therapies, which provides scientific recommendations on the classification of all advanced therapy medicinal products (ATMPs), including gene therapies, during their development, provides scientific advice on ATMP programs, and reviews ATMP dossiers.

The EU guidelines on Environmental Risk Assessment of gene therapy investigational products also need to be considered in the context of:[3]

Expedited Approval Pathways

Gene therapy products intended to treat serious or life-threatening diseases or conditions with significant unmet medical needs may be eligible for expedited approval. The FDA offers the following designations or pathways:

The FDA also offers the Initial Targeted Engagement for Regulatory Advice on CBER Products (INTERACT) program for products that introduce unique challenges due to unknown safety profiles resulting from the use of complex manufacturing technologies, development of innovative devices, or cutting-edge testing methodologies.[9] This program enables sponsors to obtain advice on a wide range of development-related topics through an informal, non-binding consultation with CBER (Center for Biologics Evaluation and Research) staff.

In the EU, the primary path to expedited approval is the Priority Medicines (PRIME) designation. Other pathways include accelerated assessment and conditional approval.

Companion diagnostics

For certain gene therapy programs, the FDA recommends the development of companion diagnostic (CDx) assays to assist in the identification of those patients who are most likely to benefit from or experience adverse events related to the investigational gene therapy. CDx may also be useful for confirming the genetic defect targeted by the gene therapy product or for assessing the therapeutic potential of the gene therapy product.[10] If it is expected that a CDx will ultimately be used for patient selection in clinical practice, sponsors should coordinate submission of the gene therapy BLA and the CDx 510(k) so that marketing authorizations for both would be obtained at the same time.[11]

Study Considerations

Study design

Requirements for gene therapy clinical trial design will vary by study phase and indication. Natural history studies may be important for understanding the genotype-phenotype relationship. Generally, first-in-human (FIH) studies for gene therapy programs require staggering of enrollment and dosing to monitor safety. For rare diseases treated with gene therapies, the FDA recommends that the sponsor explores whether the FIH studies could be randomized controlled trials to generate the safety and efficacy data necessary to support registration. Historical controls can only be used if they are accompanied by knowledge of the natural history of the disease, which may be lacking in rare diseases.[11]

If the study includes a sham procedure, it is essential to not only demonstrate that this procedure does not cause undue risk or burden, but also ensure that the participant understands they may not receive the investigational gene therapy product. Country-specific regulations and requirements for sham procedures may differ and may require extra steps for EC approval. Moreover, if devices or specialized equipment are used in vector or cell administration, additional approvals including such local approvals as CE marking are needed. Overlooking these requirements can result in study delays, so planning is key.

Depending on its vector and genome editing potential, an investigational gene therapy product may require long-term follow up (LTFU) of up to 15 years. The need for LTFU presents a significant challenge to patient and site engagement and should be considered carefully in the process of study design. Establishing patient registries and creating mobile applications can be invaluable in collecting patient-reported outcomes and minimizing data loss during the prolonged follow-up period.

If the study involves pediatric patients, retention may be an even bigger hurdle as patients may relocate or mature to being followed by an adult physician who is not affiliated with the trial. To address this, sponsors may need to qualify, initiate, and train new sites during the LTFU period.

Site selection

Due to the complexity involved in gene therapy administration, it is paramount for sites to be experienced with gene therapy, familiar with the therapeutic area, and accessible to eligible patients. Sites also need appropriate facilities and procedures for receiving, storing, and preparing the gene therapy product. To the extent possible, aligning protocol requirements with existing site workflow and policies will help to limit site burden and facilitate staff engagement.

When qualifying sites, it may be useful to ask the following questions:

Relevant experience with the mode of administration is especially important in autologous gene therapy studies or trials that require an intracranial procedure.

Every study is unique. Even if a site has previous experience, it is important for sponsors to establish processes for training and supporting all sites to optimize performance. Training for site staff should include guidance on how to address GMO-related questions and concerns that patients and families may have. Performing a dry run of the protocol requirements at the site initiation visit may help reduce errors, identify risk mitigation tactics, and increase the confidence of site staff.

Recruitment and retention

As many investigational gene therapies target rare diseases, these trials face the same challenges with recruitment as other rare disease studies. Seeking and incorporating patient and caregiver feedback into the trial protocol helps ensure feasibility of the study design. That feedback helps validate that the study will be of interest and does not create excess burden to patients or their families. Sponsors may find it useful for patient support and advocacy groups to use in increasing awareness of the study through their networks.

Many patients may be unfamiliar with gene therapy. Consequently, education and informed consent are essential to successful enrollment in gene therapy trials. Setting appropriate expectations, particularly in studies that involve a sham procedure, and providing information on the risks and complicated logistics associated with gene therapy can help to alleviate concerns.

To ensure successful recruitment and retention, sponsors should focus on minimizing study-related burden and enabling participation, even under extraordinary circumstances. This is true for any clinical trial but is particularly important in gene therapy studies that require long-term follow-up. For patients who are traveling long distances, coordinating transportation and lodging can minimize the stress of site visits. If cross-border enrollment is expected, it is critical to implement processes and procedures that ensure seamless transitions.

Implementing a hub and spoke model, in which gene therapy administration occurs at a centralized, specialized location and follow-up is performed locally, may increase the accessibility and diversity of a study and facilitate enrollment. To the extent possible to do so without compromising data quality, opportunities to incorporate mobile research nursing visits and integrate technology for remote data capture should not be overlooked. Leveraging any combination of these strategies to minimize burden may not only boost enrollment and enhance retention, but also improve the overall study experience for patients and their families.

Throughout the duration of the study, it may also be useful to communicate regularly with patients and caregivers. Providing educational materials or routine updates on study progress will foster a sense of belonging and encourage engagement. These communications are a powerful reminder to patients and their families that, by participating in the study, they are part of a larger effort to advance scientific knowledge and bring novel therapies to those who need them most.

Conclusion

In 2020, the FDA received more than 900 new investigational gene or cell therapy drug applications.[11] By the year 2025, the agency anticipates that 10 to 20 gene therapies will be approved per year.[12] Gene therapy sponsors need to consistently meet the challenge of conducting safe, ethical, patient-focused studies in a dynamic regulatory and clinical trial environment. To successfully navigate the journey to marketing authorization, sponsors must understand and plan for all of the nuances associated with designing and executing these complex studies.

[1] American Society of Gene + Cell Therapy and Informa Pharma Intelligence. Gene, Cell & RNA Therapy Landscape, Q3 2021 Quarterly Data Report. Published October 2021. Available at https://asgct.org/global/documents/asgct-pharma-intelligence-quarterly-report-q3-2021.aspx.

[2] Food and Drug Administration. Cellular & Gene Therapy Guidances, as of December 10, 2021. Available at https://www.fda.gov/vaccines-blood-biologics/biologics-guidances/cellular-gene-therapy-guidances.

[3] Official Journal of the European Union. Directive 2009/41/EC of the European Parliament and of the Council of 6 May 2009 on the contained used of genetically modified micro-organisms. Available at https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32009L0041.

[4] U.S. Food and Drug Administration. Fast Track. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track. Last updated 4 January 2018.

[5] U.S. Food and Drug Administration. Breakthrough Therapy. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy. Last updated 4 January 2018.

[6] U.S. Food and Drug Administration. Priority Review. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review. Last updated 4 January 2018.

[7] U.S. Food and Drug Administration. Priority Review. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/accelerated-approval. Last updated 4 January 2018.

[8] U.S. Food and Drug Administration. Regenerative Medicine Advanced Therapy Designation. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation.

[9] U.S. Food and Drug Administration. INTERACT Meetings. https://www.fda.gov/vaccines-blood-biologics/industry-biologics/interact-meetings.

[10] US Food and Drug Administration. Human Gene Therapy for Rare Diseases: Draft Guidance for Industry. https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM610802.pdf.

[11] Cell Culture DISH. A Remarkable Year for Gene Therapies. https://cellculturedish.com/remarkable-year-for-gene-therapies/.

[12] U.S. Food and Drug Administration. Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics.

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Advancing Gene Therapy Development: Key Regulatory and Clinical Trial Considerations - PharmaLive

The Gene Therapy Market research provides a clear understanding of the markets major geographies, and also the key segments and sub-segments. The study focuses on the state of regional development, including sales volume, cost, and growth volume. The report also gives detailed company descriptions of leading players in the Gene Therapy industry that are included in Gene Therapy Market. The research investigates all of the segments based on various parameters such as market dominance, volume, and CAGR. On the fact that it is based, revenue, and sales in the Gene Therapy market, the analysts have also thoroughly examined different regions such as North America, Europe, and the Asia Pacific. For this report on the Gene Therapy market, the researchers employed extensive mixed research approaches and technologies.

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Gene Therapy Market will generate new growth opportunities 2022-2028 | GlaxoSmithKline, Bluebird Bio, Adaptimmune Therapeutics - Digital Journal

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Gene Therapy for Age-related Macular Degeneration Market Size & Analysis By 2022 -2029 RetroSense Therapeutics, REGENXBIO, AGTC ChattTenn Sports...

DUBLIN, March 21, 2022 /PRNewswire/ -- The "Cell and Gene Therapy Bioassay Services Market by Type of Therapy, Therapeutic Area, Scale of Operation and Geography: Industry Trends and Global Forecasts, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the current landscape and the likely future potential of cell and gene therapy bioassay service providers, over the next decade. The study also features an in-depth analysis, highlighting the capabilities of various industry stakeholders engaged in this field.

Given the various advantages of cell and gene therapies and their ability to address the underlying causes of serious clinical conditions, the preference for such therapeutic modalities has increased over the years. As a result, several cell and gene therapies have been developed and approved for the treatment of a wide range of disease indications. In fact, at the time of this report's publication, more than 30 such therapies had been approved and over 1,200 therapeutic leads are under evaluation in different stages.

Owing to their rising popularity, these upcoming advanced therapeutic products are on the verge of becoming one of the highest valued therapeutic segments within the biopharmaceutical industry. Having said that, as is the case for all biological products, the development of cell and gene therapies is a complex, challenging and cost intensive process. Outsourcing is, therefore, a preferred operational model, with cell and gene therapy innovators relying heavily on specialty contract service providers to handle various aspects of their product development and manufacturing operations. In fact, over three quarters of the biopharmaceutical companies prefer to outsource their operations to the contract service providers and a large part of their expenditure goes to outsourcing services.

Amongst the various operations, the outsourcing of analytical testing services, including environment testing, bioanalytical testing, analytical testing, chemistry and stability testing, method development and validation, and product characterization, has witnessed significant rise in recent years.

The rising demand for outsourcing bioanalytical services has prompted the emergence of several contract research organizations that claim to offer bioassay services for cell and gene therapies. These companies usually have relatively more experience and are well equipped with the instruments and technological platforms to offer both pre-developed and customized bioassays.

In fact, these companies assist in bringing out the maximum efficiency of study design by offering several cost benefits and reducing the associated timelines. Given the benefits of outsourcing the bioassay services and the ongoing efforts of service providers to further improve / expand their respective offerings, we believe that the cell and gene therapy bioassay services market is likely to evolve at a steady pace, till 2030.

In addition to other elements, the study includes:

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

4. MARKET LANDSCAPE

5. COMPANY COMPETITIVENESS ANALYSIS

5.1. Chapter Overview

5.2. Methodology

5.3. Key Parameters

5.4. Competitiveness Analysis: Companies Offering Cell and Gene Therapy Bioassay Services in North America

5.5. Competitiveness Analysis: Companies Offering Cell and Gene Therapy Bioassay Services in Europe and Asia-Pacific

6. CELL AND GENE THERAPY BIOASSAY SERVICE PROVIDERS IN NORTH AMERICA: COMPANY PROFILES

6.1. Chapter Overview

6.2. CCRM

6.2.1. Company Overview

6.2.2. Cell and Gene Therapy Bioassay Service Portfolio

6.2.3. Recent Developments and Future Outlook

6.3. Nexelis

6.3.1. Company Overview

6.3.2. Cell and Gene Therapy Bioassay Service Portfolio

6.3.3. Recent Developments and Future Outlook

6.4. Pacific BioLabs

6.4.1. Company Overview

6.4.2. Cell and Gene Therapy Bioassay Service Portfolio

6.4.3. Recent Developments and Future Outlook

6.5. PPD Laboratories

6.5.1. Company Overview

6.5.2. Cell and Gene Therapy Bioassay Service Portfolio

6.5.3. Recent Developments and Future Outlook

6.6. WuXi Advanced Therapies

6.6.1. Company Overview

6.6.2. Cell and Gene Therapy Bioassay Service Portfolio

6.6.3. Recent Developments and Future Outlook

7. CELL AND GENE THERAPY BIOASSAY SERVICE PROVIDERS IN EUROPE AND ASIA-PACIFIC: COMPANY PROFILES

7.1. Chapter Overview

7.2. AnaBioTec

7.2.1. Company Overview

7.2.2. Cell and Gene Therapy Bioassay Service Portfolio

7.2.3. Recent Developments and Future Outlook

7.3. Intertek Pharmaceutical Services

7.3.1. Company Overview

7.3.2. Cell and Gene Therapy Bioassay Service Portfolio

7.3.3. Recent Developments and Future Outlook

7.4. Lonza

7.4.1. Company Overview

7.4.2. Cell and Gene Therapy Bioassay Service Portfolio

7.4.3. Recent Developments and Future Outlook

7.5. Porton Bio

7.5.1. Company Overview

7.5.2. Cell and Gene Therapy Bioassay Service Portfolio

7.5.3. Recent Developments and Future Outlook

8. MARKET TREND ANALYSIS

9. GLOBAL EVENT AND STRATEGIC INITIATIVE ANALYSIS

10. CASE STUDY: MARKET LANDSCAPE OF CELL AND GENE THERAPIES

10.1. Chapter Overview

10.2. Cell Therapy Market

10.2.1. T-Cell Immunotherapies: Development Pipeline

10.2.1.1. Analysis by Type of Product

10.2.2. CAR-T Cell Therapies: Development Pipeline

10.2.2.1. Analysis by Phase of Development

10.2.2.2. Analysis by Therapeutic Area

10.2.2.3. Analysis by Type of Developer

10.2.2.4. Key Industry Players: Analysis by Number of CAR-T Cell Therapies in Development

10.2.3. TCR-based Therapies: Development Pipeline

10.2.3.1. Analysis by Phase of Development

10.2.3.2. Analysis by Therapeutic Area

10.2.3.3. Analysis by Type of Developer

10.2.3.4. Key Industry Players: Analysis by Number of TCR-based Therapies in Development

10.2.4. TIL-based Therapies: Development Pipeline

10.2.4.1. Analysis by Phase of Development

10.2.4.2. Analysis by Therapeutic Area

10.2.4.3. Analysis by Type of Developer

10.2.4.4. Key Industry Players: Analysis by Number of TIL-based Therapies in Development

10.3. Gene Therapy Market

10.3.1. Gene Therapy Market: Clinical and Commercial Pipeline

10.3.1.1. Analysis by Phase of Development

10.3.1.2. Analysis by Therapeutic Area

10.3.2. Gene Therapy Market: Development Pipeline

10.3.2.1. Analysis by Phase of Development

10.3.2.2. Analysis by Therapeutic Area

11. LIKELY PARTNERS FOR CELL THERAPY BIOASSAY SERVICE PROVIDERS

11.1. Chapter Overview

11.2. Scope and Methodology

11.3. Potential Strategic Partners for Cell Therapy Bioassay Service Providers

11.3.1. Likely Partner Opportunities for Stem Cell Therapy Bioassay Service Providers

11.3.2. Likely Partner Opportunities for CAR-T Therapy Bioassay Service Providers

11.3.3. Likely Partner Opportunities for Dendritic Cell Therapy Bioassay Service Providers

11.3.4. Likely Partner Opportunities for Tumor Cell Therapy Bioassay Service Providers

11.3.5. Likely Partner Opportunities for NK Cell Therapy Bioassay Service Providers

12. LIKELY PARTNERS FOR GENE THERAPY BIOASSAY SERVICE PROVIDERS

12.1. Chapter Overview

Excerpt from:
Global Cell and Gene Therapy Bioassay Services Market to 2030 - by Type of Therapy, Therapeutic Area, Scale of Operation and Geography - 69News...

PHILADELPHIA (KYW Newsradio) For more than 40 years, researchers have been looking for a way to cure HIV. Now, that solution may finally be just around the corner.

Temple University researchers launched a clinical trial last month. The treatment removes HIV from infected cells using gene-editing technology, a therapy known as CRISPR.

When you do this CRISPR cleavage, you basically cut the virus on two ends, explained Dr. Tricia Burdo, associate professor in the Department of Microbiology, Immunology and Inflammation at Temples School of Medicine.

You cut a really big piece out, she said. The body has this natural ability to repair its own DNA, so once you remove that HIV from the DNA, your DNA will then come back together and repair itself.

That cut isnt made with a knife or scissors, but rather with molecules and proteins that enter the body during an infusion, which takes about an hour and a half.

Dr. Kamel Kalili, director of Temples Center for Neurovirology and Gene Editing, said the trials were done with animals.

The result of the intravenous inoculation of the CRISPR result in complete elimination of the virus from every single organ that we examined, he said.

And just a few weeks after the infusion, the virus was gone.

Clinical trials on humans are ongoing.

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Temple researchers using gene therapy to find a cure for HIV - KYW

DUBLIN--(BUSINESS WIRE)--The "Cell and Gene Therapy Bioassay Services Market by Type of Therapy, Therapeutic Area, Scale of Operation and Geography: Industry Trends and Global Forecasts, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the current landscape and the likely future potential of cell and gene therapy bioassay service providers, over the next decade. The study also features an in-depth analysis, highlighting the capabilities of various industry stakeholders engaged in this field.

Given the various advantages of cell and gene therapies and their ability to address the underlying causes of serious clinical conditions, the preference for such therapeutic modalities has increased over the years. As a result, several cell and gene therapies have been developed and approved for the treatment of a wide range of disease indications. In fact, at the time of this report's publication, more than 30 such therapies had been approved and over 1,200 therapeutic leads are under evaluation in different stages.

Owing to their rising popularity, these upcoming advanced therapeutic products are on the verge of becoming one of the highest valued therapeutic segments within the biopharmaceutical industry. Having said that, as is the case for all biological products, the development of cell and gene therapies is a complex, challenging and cost intensive process. Outsourcing is, therefore, a preferred operational model, with cell and gene therapy innovators relying heavily on specialty contract service providers to handle various aspects of their product development and manufacturing operations. In fact, over three quarters of the biopharmaceutical companies prefer to outsource their operations to the contract service providers and a large part of their expenditure goes to outsourcing services.

Amongst the various operations, the outsourcing of analytical testing services, including environment testing, bioanalytical testing, analytical testing, chemistry and stability testing, method development and validation, and product characterization, has witnessed significant rise in recent years.

The rising demand for outsourcing bioanalytical services has prompted the emergence of several contract research organizations that claim to offer bioassay services for cell and gene therapies. These companies usually have relatively more experience and are well equipped with the instruments and technological platforms to offer both pre-developed and customized bioassays.

In fact, these companies assist in bringing out the maximum efficiency of study design by offering several cost benefits and reducing the associated timelines. Given the benefits of outsourcing the bioassay services and the ongoing efforts of service providers to further improve / expand their respective offerings, we believe that the cell and gene therapy bioassay services market is likely to evolve at a steady pace, till 2030.

Companies Mentioned

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

4. MARKET LANDSCAPE

5. COMPANY COMPETITIVENESS ANALYSIS

6. CELL AND GENE THERAPY BIOASSAY SERVICE PROVIDERS IN NORTH AMERICA: COMPANY PROFILES

7. CELL AND GENE THERAPY BIOASSAY SERVICE PROVIDERS IN EUROPE AND ASIA-PACIFIC: COMPANY PROFILES

8. MARKET TREND ANALYSIS

9. GLOBAL EVENT AND STRATEGIC INITIATIVE ANALYSIS

10. CASE STUDY: MARKET LANDSCAPE OF CELL AND GENE THERAPIES

11. LIKELY PARTNERS FOR CELL THERAPY BIOASSAY SERVICE PROVIDERS

12. LIKELY PARTNERS FOR GENE THERAPY BIOASSAY SERVICE PROVIDERS

13. MARKET FORECAST AND OPPORTUNITY ANALYSIS

14. CONCLUSION

15. EXECUTIVE INSIGHTS

16. APPENDIX 1: TABULATED DATA

17. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

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Insights on the Cell and Gene Therapy Bioassay Services Market to 2030 - Industry Trends and Global Forecasts - ResearchAndMarkets.com - Business Wire

Mustang Bio, Inc.

WORCESTER, Mass., March 23, 2022 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced financial results and recent corporate highlights for the full year ended December 31, 2021.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, In 2021, we continued to make great strides in advancing our cell and gene therapy programs. Notably, the U.S. Food and Drug Administration (FDA) approved two Mustang Investigational New Drug (IND) applications, the first to initiate a pivotal multicenter Phase 2 clinical trial to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with X-linked severe combined immunodeficiency (XSCID), and the second to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106, a CD20-targeted autologous CAR T cell therapy for relapsed or refractory B-cell non-Hodgkin lymphomas (B-NHL) and chronic lymphocytic leukemia (CLL). Additionally, we received a $2 million grant from the National Cancer Institute to partially fund our MB-106 Phase 2 clinical trial. Interim data from the ongoing Phase 1/2 clinical trial of MB-106 presented at the 63rd American Society of Hematology Annual Meeting (ASH2021) by our collaborators at Fred Hutchinson Cancer Research Center (Fred Hutch) showed a 95% overall response rate, 65% complete response rate and favorable safety profile. We also expanded our product portfolio by executing an exclusive license agreement with Leiden University Medical Centre for a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID and enhanced our CAR T program with an exclusive license agreement with Mayo Clinic for a novel technology to create in vivo CAR T cells that has the potential to transform the administration of CAR T therapies and be used as an off-the-shelf therapy.

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Dr. Litchman continued, We anticipate another productive year in 2022, with several Mustang IND clinical trial initiations and data updates from our ongoing clinical programs at prominent medical conferences. In February, City of Hope presented Phase 1 data on MB-105, our prostate stem cell antigen (PSCA) CAR T-cell therapy for the treatment of PSCA-positive metastatic castration-resistant prostate cancer (mCRPC) at the 2022 American Society of Clinical Oncology (ASCO) Genitourinary (GU) Cancers Symposium that demonstrated its potential to treat patients with mCRPC. In April, our collaborators at Fred Hutch will present interim Phase 1/2 clinical trial data on MB-106 at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR) which show its compelling clinical activity and favorable safety profile. In the first half of 2022 we anticipate enrolling the first patient in Mustangs multicenter Phase 1/2 clinical trial to evaluate MB-106, and in the second half of 2022 we expect to enroll the first patient in Mustangs multicenter pivotal Phase 2 clinical trial to evaluate MB-107. We also plan to advance additional Mustang clinical candidates, including filing an IND for MB-109 (MB-101 autologous IL13R2-directed CAR T cells + MB-108 oncolytic virus) for the treatment of glioblastoma. With this steady progress across our clinical programs, and with our robust pipeline and strong cash position following completion of the debt facility with Runway Growth Capital, Mustang is poised to continue its success building an integrated cell and gene therapy company.

Financial Results:

As of December 31, 2021, Mustangs cash and cash equivalents and restricted cash totaled $110.6 million, compared to $98.8 million as of December 31, 2020, an increase of $11.8 million year-to-date.

Research and development expenses were $49.9 million for the year ended December 31, 2021. This compares to $37.2 million for 2020. Non-cash, stock-based compensation expenses included in research and development were $2.3 million for the year ended December 31, 2021, compared to $1.4 million for 2020.

Research and development expenses from license acquisitions totaled $5.8 million for the year ended December 31, 2021, compared to $10.1 million for 2020. Non-cash, stock-based compensation expenses included in research and development licenses acquired were $4.2 million for the year ended December 31, 2021, compared to $7.6 million for 2020.

General and administrative expenses were $11.0 million for the year ended December 31, 2021. This compares to $9.5 million for 2020. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.9 million for the year ended December 31, 2021, compared to $4.0 million for 2020.

Net loss attributable to common stockholders was $66.4 million, or $0.76 per share, for the year ended December 31, 2021, compared to a net loss attributable to common stockholders of $60.0 million, or $1.14 per share, for 2020.

2021 and Recent Corporate Highlights:

In February 2021, Mustang announced encouraging MB-107 and MB-207 clinical updates from its X-linked severe combined immunodeficiency (XSCID) investigator IND trials, as well as additional consistent safety and efficacy data. In the second half of 2022, Mustang expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial under Mustang Bios IND to evaluate MB-107, a lentiviral gene therapy for the treatment of infants under the age of two with XSCID, also known as bubble boy disease. Mustang filed an IND application in December 2021 for its pivotal multicenter Phase 2 clinical trial of MB-207, a lentiviral gene therapy for the treatment of patients with XSCID who have been previously treated with a hematopoietic stem cell transplantation (HSCT) and for whom re-treatment is indicated. The trial is currently on hold pending CMC clearance from FDA and, based on feedback from the Agency, Mustang expects to enroll the first patient in a pivotal multicenter Phase 2 clinical trial in the first quarter of 2023.

In May 2021, Mustang announced that the FDA approved its IND application to initiate a multicenter Phase 1/2 clinical trial investigating the safety and efficacy of MB-106, a CD20-targeted autologous CAR T cell therapy for relapsed or refractory B-NHL and CLL. Mustang intends to dose the first patient in that trial in the first half of 2022.

Also in May 2021, Mustang announced that the first patient was dosed at City of Hope in a clinical trial to establish the safety and feasibility of administering MB-101 (autologous IL13R2-directed CAR T cells) to patients with leptomeningeal brain tumors (e.g., glioblastoma, ependymoma or medulloblastoma).

In June 2021, Mustang announced that MB-106 CD20-targeted CAR T cell therapy data were presented at the European Hematology Association 2021 Virtual Congress. Dr. Mazyar Shadman of Fred Hutch presented updated interim data from the ongoing Phase 1/2 clinical trial for B-NHL and CLL, which showed a favorable safety profile and compelling clinical activity, with a 93% overall response rate and 67% complete response rate in patients treated with a modified cell manufacturing process.

Also in June 2021, Mustang hosted a key opinion leader webinar featuring a presentation from Dr. Shadman, who discussed interim results from the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted CAR T for B-NHL and CLL.

Additionally in June 2021, Mustang announced that it was one of 28 recipients awarded tax incentives from the Massachusetts Life Sciences Center. The $300,000 tax incentive amount that Mustang was awarded was based on a hiring commitment of 20 net new full-time equivalent employees for calendar year 2021 and retaining that headcount level through 2025.

In August 2021, Mustang announced that the European Medicines Agency granted Priority Medicines (PRIME) designation to MB-107, a lentiviral gene therapy for the treatment of XSCID in newly diagnosed infants.

Also in August 2021, Mustang announced an exclusive license agreement with Mayo Clinic for a novel technology to create in vivo CAR T cells that may be able to transform the administration of CAR T therapies and has the potential to be used as an off-the-shelf therapy.

In October 2021, Christine Brown, Ph.D., Deputy Director, T Cell Therapeutics Research Laboratory and The Heritage Provider Network Professor in Immunotherapy at City of Hope, presented updated Phase 1 clinical data regarding MB-101 (IL13R2targeted CAR T cells) for the treatment of glioblastoma at two scientific conferences, the First Annual Conference on CNS Clinical Trials, co-sponsored by the Society for Neuro-Oncology and American Society of Clinical Oncology, and the American Association for Cancer Research Virtual Special Conference: Brain Cancer.

In November 2021, Mustang announced the execution of an exclusive license agreement with Leiden University Medical Centre for a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID.

Also in November 2021, Mustang announced that it was awarded a grant of approximately $2 million from the National Cancer Institute of the National Institutes of Health. This two-year award will partially fund the Mustang-sponsored multicenter trial to assess the safety, tolerability and efficacy of MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell NHL or CLL.

In December 2021, Mustang announced that MB-106 data were presented at ASH2021. Dr. Shadman of Fred Hutch presented updated interim data showing a 95% overall response rate, 65% complete response rate and favorable safety profile from the ongoing Phase 1/2 clinical trial for B-NHL and CLL. A copy of the poster can be viewed online here.

Also in December 2021, Mustang hosted a key opinion leader webinar featuring a presentation from Dr. Shadman, who discussed interim results from the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted, autologous CAR T cell therapy to treat B-NHL and CLL. A replay of the webinar can be found here.

Additionally in December 2021, Mustang was added to the NASDAQ Biotechnology Index.

In January 2022, Mustang announced that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell NHL and CLL, were selected for a poster presentation at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the ASTCT and CIBMTR, rescheduled to take place April 23-26, 2022, in Salt Lake City, Utah. A copy of the abstract can be viewed on the meeting website here.

In February 2022, Mustang was selected as the Bronze winner for the Central region in the Eighteenth Annual Team Massachusetts Economic Impact Awards presented by MassEcon. The award winners will be honored at Gillette Stadium on April 7, 2022.

Also in February 2022, Phase 1 data on MB-105, a PSCA-targeted CAR T cell therapy administered systemically to patients with PSCA-positive mCRPC, were presented by City of Hope at the 2022 American Society of Clinical Oncology Genitourinary Cancers Symposium. The data results indicated that PSCA-CAR T-cell therapy is feasible in patients with mCRPC with dose limiting toxicity (DLT) of cystitis and show preliminary anti-tumor effect at a dose of 100 million cells plus lymphodepletion. It was concluded that escalation up to the next dose level of 300 million cells can proceed in the trial.

In March 2022, Mustang completed a $75 million long-term debt facility with Runway Growth Capital LLC ("Runway).

Also in March 2022, Mustang announced that an abstract reporting on Phase 1 trials being conducted at the University of Alabama at Birmingham (UAB) and City of Hope of Mustang Bios exclusively licensed oncolytic viral and CAR T-cell therapies for the treatment of patients with glioblastoma (GBM) was selected as a late-breaking poster presentation at the American Association for Cancer Research (AACR) Annual Meeting 2022, taking place April 8 13, 2022, in New Orleans, Louisiana. The abstract will also be published in the online Proceedings of the AACR.

About Mustang BioMustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR-T therapies across multiple cancers, as well as lentiviral gene therapies for severe combined immunodeficiency. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

MUSTANG BIO, INC.Balance Sheets (in thousands, except for share and per share amounts)

December 31,

December 31,

2021

2020

ASSETS

Current Assets:

Cash and cash equivalents

$

109,618

$

97,804

Other receivables - related party

50

15

Prepaid expenses and other current assets

2,038

1,715

Total current assets

111,706

99,534

Property, plant and equipment, net

9,025

7,529

Fixed assets - construction in process

2,027

499

Restricted cash

1,000

1,000

Other assets

362

250

Operating lease right-of-use asset, net

1,050

1,088

Total Assets

$

125,170

$

109,900

LIABILITIES AND STOCKHOLDERS EQUITY

Current Liabilities:

Accounts payable and accrued expenses

$

9,744

$

8,747

Payables and accrued expenses - related party

723

490

Operating lease liabilities - short-term

348

278

Total current liabilities

10,815

9,515

Deferred income

270

Operating lease liabilities - long-term

1,685

1,950

Total Liabilities

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Mustang Bio Reports Full-Year 2021 Financial Results and Recent Corporate Highlights - Yahoo Finance

"Throughout his career, Rayne has demonstrated strong leadership in multiple roles across the globe, including most recently as chief manufacturing officer leading the development of our state-of-the-art manufacturing facility in Thousand Oaks," said Peter Anastasiou, president and CEO of Capsida. "Rayne's appointment to COO will ensure Capsida achieves its goal of moving our technology from the lab and to patients, and will help Capsida realize the promise of gene therapy."

"I'm honored to take on the role of COO at Capsida, a company that always puts patients first," said Mr. Waller. "Throughout my career in the biotechnology industry, I've worked to focus on the end goal of bringing therapies to patients who have no other options. As COO, I look forward to working with the Capsida leadership team and our board to bring the promise of gene therapy to life."

Gene therapy is still in its infancy and has yet to achieve its full potential. Capsida's proprietary, targeted, non-invasive gene therapy technology allows more selective targeting of specific tissues and cells, overcoming many of the problems associated with first-generation gene therapies, specifically off-target cell and organ activity. In addition, it allows the gene therapy to be delivered non-invasively through intravenous administration.

Prior to joining Capsida, Mr. Waller spent 27 years at Amgen in increasing roles of responsibility across manufacturing and supply-chain management. His most recent roles at Amgen included vice president and site head for Amgen's largest manufacturing site in Puerto Rico, vice president of regional manufacturing, responsible for overseeing site operations for manufacturing facilities in Ireland and The Netherlands, and vice president of global supply-chain management with responsibility for contract manufacturing, global supply management, operations strategic planning, and risk management.

Mr. Waller holds a bachelor's degree in business administration from the University of Arizona.

About Capsida Biotherapeutics

Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of rare and more common genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology. Visit us atwww.capsida.comto learn more.

SOURCE Capsida Biotherapeutics

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Capsida Biotherapeutics Announces Appointment of Rayne Waller to Chief Operating Officer - PR Newswire

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You don't go to the leading edge of life sciences/bioproduction looking for bargains, as the strong growth and margins available to companies facilitating the rapid growth of new biologic treatment options like gene and cell therapies have fueled strong share price performance (and multiples) for larger, better-known companies like Danaher (DHR) and Thermo Fisher (TMO), as well as smaller players like Bio-Techne (NASDAQ:TECH).

I find a lot to like in Bio-Techne's leverage to cell and gene therapy-enabling products like GMP protein production and non-viral gene editing, as well as its leverage to life sciences/bioproduction research tools, spatial biology, and molecular diagnostics. I don't find nearly as much to like in the valuation, but stocks like these are a "you either get it, or you don't" sort of proposition where you're basically betting that the underlying growth of the market and the company's strategic decisions will eventually lead to enough revenue and profit growth down the line to redeem an eye-watering valuation today.

Through both M&A and leveraging internal manufacturing capabilities grounded in a long history of producing proteomic research reagents, Bio-Techne has built a business segment to leverage the exceptional underlying growth potential in cell and gene therapies over the coming years.

Bio-Techne's offerings include Cloudz cell activation kits that use non-magnetic beads to separate and activate cells, the TcBuster non-viral gene editing platform, and its GMP proteins, as well as complementary tools in spatial biology and immunocytochemistry. All told, management has sized its opportunity in CGT at over $3B today, growing at over 20%, and the company only has a small low-single-digit market share today.

GMP proteins are a major thematic driver right now, growing 180% in the fourth quarter and helping drive 80%-plus growth overall for the Cell and Gene Therapy (or CGT) business. GMP proteins are proteins produced in dedicated facilities and in accordance with strict quality management guidelines, ensuring biological activity, purity, and batch-to-batch consistency.

These proteins are not used directly in therapies (they're not infused or injected into patients), but they're used to produce cell and gene therapies that are practically in their infancy. As such, I think a handy shortcut for readers is to think of them as consumables used in the development and manufacture of cell and gene therapies like Novartis' (NVS) Kymriah (CAR-T) and Zolgensma (gene therapy).

Management recently opened a new GMP protein manufacturing facility and the ramp of commercial operations there should drive meaningful growth for several quarters, with the business scaling from around $30M to over $200M over the next several years. In addition to ramping up this facility, management has already started talking of broadening the business into GMP antibodies and media, as well as RNA and DNA products. As an aside, Bio-Techne tried to acquire Aldevron, a manufacturer of GMP-grade plasmid DNA, mRNA, and proteins back in 2021 but lost out to Danaher.

Bio-Techne's CGT operations are the "sizzle" today, but there's plenty of steak behind it. While the company's legacy proteomic reagents and diagnostics (calibrators and controls) operations aren't particularly sexy, they are still growing at a mid-single-digit clip and they generate good cash flow for the business.

Beyond this are interesting opportunities in analytical instrumentation, spatial biology, and molecular diagnostics.

The Analytical Solutions business is growing at a double-digit rate for Bio-Techne, with the company leveraging growing demand for automation in the lab. The Simple Western is the only automated tool for western blotting, a technique that identifies specific proteins in a sample and can require hours of hands-on time. The Maurice platform automates protein profiling while Ella, a benchtop immunoassay platform, can be used in a range of applications like monitoring cytokine storms (in CAR-T recipients or COVID-19 patients) and detecting impurities in finished biologics production runs.

Bio-Techne's Advanced Cell Diagnostics business offers RNAscope and DNAscope, and an in-situ RNA/DNA hybridization platform that detects target RNA or DNA within a single cell without disturbing the tissue morphology. This allows users to examine biomarker status, structural variation, and so on, and is useful in a range of applications from cancer diagnostics to ensuring whether reprogrammed T-cells for CAR-T are expressing the intended antigen receptors.

In molecular diagnostics, Bio-Techne hasn't seen the hoped-for adoption of its ExoDx Prostate IntelliScore liquid biopsy for prostate cancer (a rule-out test after ambiguous PSA results), but reimbursement has improved and post-pandemic normalization of office visits should drive more use. Beyond this, though, the company is building out a broader portfolio of biomarker-based screening products that can address a range of diseases/conditions, with a particular focus on genetic carrier screening and oncology diagnostics.

All told, Bio-Techne is targeting markets with combined addressable revenue of over $15 billion a year, including high-growth areas like analytical research tools, cell/gene therapy, spatial biology, liquid biopsy, and molecular diagnostics. Given that cell and gene therapies are practically in their infancy (or at least early toddlerhood), I see significant growth opportunities in tools and consumables that facilitate research and production, and I think Bio-Techne has a good starting position today.

I also doubt that the company is anywhere close to done with its M&A program. More than a decade ago, I followed Bio-Techne (then Techne) as a sell-side analyst, and the change in the company since then has been remarkable. Much of that change can be tied to a change in management and operating philosophy (actively targeting growth), but M&A has played a key enabling role, including deals for Asuragen (diagnostics kitting), B-MoGen (TcBuster gene editing), Quad Technologies (Cloudz), Exosome, CyVek (Ella) and ProteinSimple (automated equipment, including Simple Western).

Most recently, the company announced an unusual deal for Wilson Wolf, the manufacturer of G-Rex bioreactors (used to grow/produce proteins or cells like T-cells in volume). Bio-Techne isn't buying the company today but now has the option to buy 20% when the company reaches $100M in revenue and the whole company at $225M in revenue (or $135M in EBITDA), as well as another option if those targets aren't reached. No definitive terms have been reached, but at $225M in revenue, it seems likely that the deal would cost over $2B.

I'm looking for high-teens annualized revenue growth over the next decade and EBITDA margin expansion to over 42% in 2024 (as well as long-term FCF margins in the high 20%s), but those targets don't really matter. I don't think there's any way to derive a bottoms-up fair value that will look attractive today, though comparative analysis to other high-growth life sciences/bioproduction names suggests a current EV/forward revenue of around 16.5x isn't completely unprecedented (Danaher paid 17.6x forward revenue for Aldevron).

Investing in highly-valued "hyper-growth" stocks has never really been in my wheelhouse; I've done it and made money doing it, but I'm always reluctant to make "forget the valuation and buy it'll all work out eventually" recommendations to others. I do like Bio-Techne's leverage to growth in bioproduction generally and cell/gene therapy research and production more specifically, but this is definitely not a stock for investors who can't accept high risk.

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Bio-Techne - Exceptional Leverage To Biopharma Growth, And Priced Accordingly - Seeking Alpha

Wet age-related macular degeneration (wAMD) has recently witnessed a series of approvals thanks to a degree of product differentiation, and this has drawn enthusiasm from multiple stakeholders in the field. This interest is expected to be widened with the anticipated approvals of more products, particularly biosimilars. Despite this, a lack of treatment options for other sub-types of the disease means that opportunities are still available for pharmaceutical companies to make a difference to patients affected by AMD.

Product differentiation has been a key strategy employed by companies whose products have recently received approvals for wAMD. Consider for example Roches Susvimo (ranibizumab), which received US Food and Drug Administration (FDA) approval in October last year. Patients implanted with Susvimo are expected to visit their doctor only twice yearly to have the implant refilled so that the medicine can be delivered to their eyes continuously. That represents a significant alleviation of treatment burden when compared with the current standards of care such as Lucentis (ranibizumab), Eylea (aflibercept) and Beovu (brolucizumab), which need to be administered more frequently.

Vabysmo (faricimab), also from Roche, received FDA approval in January this year for wAMD and for diabetic macular oedema. This therapy is administered every four weeks for the first four doses, following which, depending on the results of optical coherence tomography and visual acuity evaluations, there is potential for the frequency of administration to be reduced to as few as twice a year, resulting in a lower cost of treatment compared to standard of care. While it remains to be seen to what extent these new therapies will be commercially successful, being associated with lesser frequency of administration already makes such therapies an attractive option for patients and physicians in this field.

Looking ahead, anticipated launches of new biosimilars and innovator molecules are expected to widen interest in the field of AMD. According to GlobalDatas Pharmaceutical Intelligence Centre (PIC), there are currently 15 products in late-stage development exclusively for wAMD in the seven major markets (7MM: the US, France, Germany, Italy, Spain, UK and Japan). Five of these are aflibercept biosimilars and four are ranibizumab biosimilars. While both groups of biosimilars are expected to directly target patient shares from their branded counterparts, Eylea (aflibercept) and Lucentis (ranibizumab), the latter two also face threats from innovator molecules including RGX-314 from RegenxBio and AbbVie. This is a gene therapy that has the potential to be used as a one-time treatment option for patients with wAMD and, if approved, has the potential to disrupt current market dynamics for this sub-type of AMD.

In addition, there are gaps that exist in the market, particularly for therapies that can help patients with dry AMD and geographic atrophy, for which there are currently no approved therapies available. According to GlobalDatas PIC, there are three therapies in late-stage development for geographic atrophy: Alkeus Pharmaceuticals ALK-001, Apellis Pharmaceuticals pegcetacoplan, and IVERIC bios avacincaptad pegol sodium. Dobecure SLs therapy ethamsylate is also in development for both dry AMD (dAMD) and wAMD. It remains to be seen, however, how effective these therapies will be in addressing the disease at an earlier stage, as it is crucial to treat these diseases before they progress further.

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Product approvals and unmet treatment needs expected to widen interest in AMD - Pharmaceutical Technology