Global Cell & Gene Therapy Business and Investment Opportunities Q1 2023 Update: An Expanding CGT Pipeline Bodes Well for Growth

DUBLIN, Feb. 24, 2023 /PRNewswire/ -- The "Global Cell & Gene Therapy Business and Investment Opportunities - Analysis & Market Size by Technology, Clinical Trials, Patents, Financial Deals, Competitive Landscape - Q1 2023 Update" report has been added to  ResearchAndMarkets.com's offering.

The lives of people all over the world have been improved by traditional methods of patient treatment that use drugs and surgery, yet there are many diseases - possibly even most of them - for which these conventional methods only slow the progression of the illness without curing it. This flaw is especially evident when treating monogenic diseases, which are congenital illnesses brought on by single-gene abnormalities.

More than 30 million people in the United States are thought to be affected by one of the more than 7,000 rare diseases that exist. Cells and genetically modified cells are "living drugs" that may repair and replace damaged tissues or ill organs, which makes them fundamentally different from medications and surgery. Due to these characteristics, they may be used to treat a variety of conditions.

There are presently six FDA-approved CAR-T cell therapies available to patients in the United States and Europe to treat different types of blood cancers. They are one of many promising cell and gene therapies that are being developed for both rare and common disorders.

A glimmer of hope for patients waiting for long-term, potentially curative treatments is the fact that more than 1,000 clinical trials, including 200 in Phase III, are now under progress. Phase I/II combo studies are frequently used. Because it enables sponsors to improve and accelerate their clinical development programmes, this design approach is still being used more and more in the industry. The industry must overcome funding, regulatory, and legislative barriers, and many patients won't benefit unless we transform the way we provide healthcare.

An expansion of the CGT pipeline

The year 2022 marked a turning point for the licencing of never-before-approved gene treatments, with three new medications licenced to treat uncommon illnesses and another authorised to treat bladder cancer. This is just the first wave of transforming ripple. By the end of 2023, innovative cell or gene therapies may have received approval for use in Europe, the United States, or both. The industry is on the verge of achieving the FDA's widely reported 2019 forecast that 10-20 innovative cell and gene therapies will be approved annually by 2025.

• In December 2022 a chimeric antigen receptor (CAR) T-cell therapy called Yescarta (axicabtagene ciloleucel) has been approved by the Japanese Ministry of Health, Labour and Welfare (MHLW) for the initial treatment of patients with relapsed/refractory large B-cell lymphoma (R/R LBCL), including those with diffuse large B-cell lymphoma and primary mediastinal large B-cell lymphoma, transformed follicular lymphoma, and high-grade B-cell lymphoma.
• Yescarta (axicabtagene ciloleucel), a CAR T-cell treatment, was given FDA approval in April 2022 for treating adult patients with large B-cell lymphoma.
• The European Commission (EC) approved Kymriah (tisagenlecleucel), a CAR-T cell therapy, for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy, according to Novartis.
• Commercial demand for Abecma (idecabtagene vicleucel) remained strong into 2022. In addition to increasing manufacturing capacity, 2seventy bio and BMS made major advancements in key supply chain indicators.
• In 2023, the company projects topline U.S. sales of $470-$570M thanks to further expansions in the manufacturing capacity of vector and pharmacological products, including an extra adherent vector manufacturing suite. 2seventy Bio anticipates additional commercial growth with an anticipated label expansion in 2024-2025. With this expansion, 2seventybio anticipates that Abecma will produce $200-300M in operational income for 2seventybio between 2024 and 2025.
• The first cell-based gene therapy, Zynteglo (betibeglogene autotemcel), has been given the green light by the US Food and Drug Administration to treat adult and paediatric beta-thalassemia patients who need regular red blood cell transfusions

In the future, it's anticipated that M&A activity will increase

• In the field of CGT, where licencing and cooperative arrangements continue to dominate. This is hardly surprising given the relative youth of CGT technology, as there are still questions about values and technical obstacles. Partnerships give the licensor or investor a reduced risk way to increase CGT offerings without taking on full financial responsibility or experiencing the productivity disruption frequently associated with M&A.
• Investments are going into firms of all stages of development, from start-ups to significant acquisitions by big pharmaceutical firms. In 2019, to purchase Celgene, Bristol Myers Squibb paid $74 billion. The largest of them are involved in oncology treatments.
• Cell and gene therapy market draws an increased amount and share of public and private investment despite the limited number of licenced products. Despite the fact that during the past ten years, all private sector investment in the life sciences has increased significantly, the exponential growth of overall investments in cell and gene therapy firms is extraordinary.
• Neogene Therapeutics, a global clinical-stage biotechnology firm that is leading the discovery, development, and production of next-generation T-cell receptor treatments (TCR-Ts), which provide a unique cell therapy strategy for treating cancer, has announced that AstraZeneca is acquiring it.

Growing cell and gene therapy enterprises have received support from this surge in private money as they reach the stock market. Initially, it showed early growth but later it went through a period of severe distortions during the pandemic. It is expected that cell and gene firms return to a more measured growth trajectory, underpinned by a growing number of clinical milestones and perhaps more regulatory approvals, as stock markets adapt and the economy picks up through the projected period.

European region may regain its feet in the field of cell and gene therapy with regulatory checks

• Although authorities in the US and Europe are moving forward with reforming their regulatory frameworks in response to scientific advancements, barriers to patient access remain a major issue in both places.
• The European Union will update its pharmaceuticals legislation; the proposal is anticipated in the first quarter of 2023. The project will have a significant impact on whether European patients have access to advanced therapy medicinal products for years to come by balancing affordability and access, among other issues.
• Europe faces tremendous challenges, particularly when it comes to access. Having the European Medicines Agency, a top-notch regulatory organisation, and having approved the first gene therapy, Europe has been a tremendous pioneer in the sector in many ways. However, due to a combination of complex factors, Europe is already lagging on certain crucial fronts.

Regulatory trends that will shape the future of cell and gene therapy

• In the upcoming years, it is anticipated that filing application numbers would rise. The amount of requests for Breakthrough (BT) and Regenerative Medicine Advanced Therapy (RMAT) designations, which are submitted concurrently with or during an ongoing IND file, has recently increased. While the benefits of BT and RMAT designations are comparable to those of fast-track designation, these designations also call for more robust supporting documentation.
• Within the framework of the NIH Accelerating Medicines Partnership Program, the FDA recently launched a new effort. The Bespoke Gene Therapy Consortium (BGTC) was established to remove significant barriers and streamline the small-batch gene therapy research process. By giving details on basic and clinical research, manufacturing, production, and regulatory needs, the BGTC will serve as a traffic light for these therapies.
• The FDA's "INTERACT" casual meeting programme is a relatively new project. This initiative involves informal talks between the Center for Biologics Evaluation and Research (CBER) employees and researchers/sponsors who are in the pre-IND stage of development to address the concerns and demands faced by the CGT industry within current clinical guidelines. The INTERACT meetings do not charge anything under the Prescription Drug User Fee Act (PDUFA). They are given based on CBER's resources and availability, and they do not take the place of other official meetings. Therefore, it is advised to ask for a pre-IND meeting before submitting an IND to start the Phase I first-in-human investigation. This is crucial when looking for advice on the designs of toxicity study plans.
• The FDA has just launched the Gene Therapy Pilot Program, which entails giving sponsors input in real-time throughout the clinical development process. The development cycle and submission review time should be sped up by the combination of new meeting formats. Together, these programmes provide opportunities for sponsors and regulators to speak and debate technical advancements more frequently.

To ensure that patients benefit from this technology, however, there are crucial challenges that must be resolved. Industry and the healthcare sector are prepared to invest in the CGT ecosystem and have faith that long-term problems (such as costs, safety, and reimbursement) will be resolved.

Cell and gene therapy firms and their investors should understand and distinguish marketing hype from legitimate science. This is a new market with enormous potential opportunities, but it still carries many risks until it is economically established. The potential of CGT to usher in a new era of medicine has undoubtedly inspired a lot of optimism.

This report presents an in-depth analysis of the current market scenario, with an emphasis on the future trajectory of potential opportunities in the cell and gene therapy industry. In addition to a combination of exhaustive secondary research along with primary research, the research methodology leverages a proprietary predictive analytics platform to provide unbiased business intelligence and answer key questions related to the cell and gene therapy market.

It provides a detailed analysis of cell and gene therapy market dynamics, covering clinical trials, patent data, financial deals, and company profiling details. It details market opportunities and risks across key segments - by type of therapy segment, by the purpose of manufacturing, by type of cell, by product, by route of administration, delivery mode, source of cell, source of manufacturing, therapeutic class, technology, and by type of therapy. It also details market dynamics across various end-use sectors to assess emerging opportunities.

In addition, this report provides analyst commentary on key trends, drivers, strategies, innovations, and regulations in cell and gene therapy sector.

This title provides global and regional insights along with data-centric analysis for the following 6 regions and 20 countries.

Scope

Insights and data in the report have been segmented under the following six modules:

Module 1: Global Cell and Gene Therapy Funding and Investments Outlook

This module provides insights and data related to cell and gene therapy partnerships and investment (research and development, mergers and acquisitions, product development, commercialization, licensing, and manufacturing) and allows a peek into the futuristic trends of cell and gene therapy technology investment area.

Module 2: Global Cell and Gene Therapy Clinical Trial Data Assessment

This module evaluates the data available from clinical trials conducted utilising cell and gene technology, representing a bird's eye view of emerging market dynamics and risks in the cell and gene therapy sector.

Module 3: Global Cell and Gene Therapy Patent Data Analysis

This module presents an exhaustive study of cell and gene therapy patent analytics at various levels, including strategic research planning as well as analyzing their potential applications.

Module 4: Competitive Landscape

This module provides detailed information along with a scoring matrix of key companies related to the cell and gene therapy industry, including their historical performance and the latest developments.

Module 5: Global Cell and Gene Therapy Market Size and Forecast

This module provides projections on the market's development during the years 2018-2027 after the profound evaluation of market dynamics at the deeper segmentation level. This report also provides an in-depth, data-centric analysis of the global cell and gene therapy market at regional and country levels:

Cell & Gene Therapy Market Size by Different Therapy Segment, 2018-2027

• Cell and Gene Modified Cell Therapies
• Gene Therapy and Genome Therapy
• DNA & RNA Therapeutics

Cell & Gene Therapy Market Size by Purpose of Manufacturing

• Commercial
• Clinical

Cell & Gene Therapy Market Size by Product

• Abecma
• Amondys
• Breyanzi
• Carvykti
• Givlaari
• Kimmtrak
• Kymriah
• Leqvio
• Luxturna
• Onpattro
• Oxlumo
• Rethymic
• Skysona
• Stratagraft
• Tecartus
• Tegsedi
• Viltepso
• Vyondys
• Yescarta
• Zolgensma
• Zynteglo

Cell & Gene Therapy Market Size by Route of Administration

• Injectables
• Infusions
• Bioscaffolds
• Topicals

Cell & Gene Therapy Market Size by Type of Cell

• CAR-T Cell
• TCR Cell

Cell & Gene Therapy Market Size by Type of Therapy

• T-Cell Therapies
• NK Cell
• Dendritic Cell Therapies
• Stem Cell Therapies

Cell & Gene Therapy Market Size by Source of Cell

• Autologous
• Allogenic

Cell & Gene Therapy Market Size by Delivery Mode

• In vivo
• ex-vivo

Cell & Gene Therapy Market Size by Source of Manufacturing

• In-House
• Contract Manufacturing

Cell & Gene Therapy Market Size by Therapeutic Class

• Oncology
• Neurology
• Ophthalmology
• Gastro-Intestinal
• Musculoskeletal
• Others

Cell & Gene Therapy Market Size by Technology

• Viral Vector Technology
• Cell Immortalization Technology
• Genome Editing Technology
• Others

Cell & Gene Therapy Market Size by End User

• Pharmaceutical & Biotechnology Companies
• Academic & Research Institutes
• Hospital & Clinics

Cell & Gene Therapy Market Size by Regions

• North America
• U. S.
• Canada
• Mexico
• Europe
• France
• United Kingdom
• Germany
• Italy
• Netherlands
• Asia-Pacific
• China
• Japan
• Australia
• India
• South Korea
• MEA region
• Israel
• South Africa
• Turkey
• U.A. E
• Saudi Arabia
• Latin America
• Brazil
• Argentina
• Colombia

Key Companies Covered

• Gilead Company
• Dendreon Corporation
• Novartis
• Organogenesis Incorporated
• Vericel Corp.
• Kolon Life Science
• Bristol Myers Squibb
• bluebird bio
• Johnson & Johnson
• Juno Therapeutics

Module 6 Regulatory Outlook

This module contains a detailed understanding of the ethical, legal, and social implications of cell and gene therapy technology.

For more information about this report visit https://www.researchandmarkets.com/r/l33sji-cell-and?w=5

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