Market Challenges And Opportunities

Global Cell and Gene Therapy Market – Driver

Increasing collaboration between the key market players to develop new gene therapies

The key market players are focused on making collaborations with other companies to develop new gene therapies and commercialize those in the cell and gene therapy market. For instance, on February 15, 2023, Charles River Laboratories International, Inc., a pharmaceutical company, and Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration. Under the collaboration, the gene therapy platform targeting renal diseases, using Charles River’s established plasmid platform, eXpDNA, and decades of experience at the company’s plasmid DNA manufacturing center of excellence.

Furthermore, in April 2022, Labcorp, a leading global life sciences company, announced that it had collaborated with Xcell Biosciences, Inc., a leading developer of cell and gene therapy technologies, to advance critical work that helps clients effectively bring innovative cell and gene therapies to market. Under this collaboration, Labcorp and Xcell Biosciences, Inc. will work on a series of projects focused on improving the safety and efficacy of cell and gene therapies (CGTs), thereby strengthening Labcorp’s commitment to growing its global, comprehensive suite of CGT capabilities, and it supports pharmaceutical and biotechnology companies’ efforts to expedite therapeutic development.

Global Cell and Gene Therapy Market – Impact of Coronavirus (COVID-19) Pandemic

Since the COVID-19 virus outbreak in December 2019, the disease has spread to over 100 countries across the globe, and the World Health Organization declared it a public health emergency on January 30, 2020.

The COVID-19 pandemic had mixed impact on the global cell and gene therapy market, owing to the increased burden on hospitals and healthcare professionals during COVID-19. For instance, in March 2021, Blood cancer U.K., a U.K.-based charity organization, conducted a virtual survey in which 47% of respondents reported that their blood cancer appointments and treatment had been impacted in some manner by the pandemic whereas 45% had not been performed due to appointment cancellations or postponement.

On the other hand, the increasing conduction of clinical trials using cell and gene therapies studied globally for the treatment of COVID-19 propelled the market growth during the pandemic. For instance, according to the Coronavirus Treatment Acceleration Program (CTAP) created by the U.S. Food and Drug Administration, as of October 31, 2021, more than 60 cell and gene therapies were studied globally for the treatment of COVID-19. The cell therapy product includes cellular immunotherapies and other types of both autologous and allogeneic cells, such as stem cells and related products.

Global Cell and Gene Therapy Market: Key Developments

In January 2022, Amgen, a U.S. based multinational biopharmaceutical company, and Generate Biomedicines, a therapeutics company, announced a research collaboration agreement to discover and create protein therapeutics for five clinical targets across several therapeutic areas and multiple modalities. As part of the research agreement collaboration, Amgen would pay US$ 50 billion in upfront funding for the initial five programs with a potential transaction value of US$ 1.9 billion plus future royalties. For each program, Amgen would pay up to US$ 370 billion in future milestones.

In June 2021, CyGenica, a biotechnology company, announced that it had raised US$ 1.4 billion in a seed fund investment round, led by the global venture capital investor SOSV, a non-governmental organization. The funding round would accelerate the validation of CyGenica's proprietary technology for safe, targeted, and affordable intercellular drug delivery. The investment would accelerate the development of disruptive drug delivery technology in the advancement of therapeutics for cancer and rare diseases to improve patients’ health and quality of life.

In March 2021, Amgen Inc., a U.S. based multinational biopharmaceutical company, and Rodeo Therapeutics Corporation, a biotechnology company, announced an agreement under which Amgen would acquire Rodeo, which develops small-molecule therapies designed to promote regeneration and repair of multiple tissues. The agreement would help the company in expansion to develop therapeutics for patients using Rodeo's 15-PGDH (a prostaglandin-degrading enzyme) program with Amgen's inflammation portfolio.

In March 2021, the U.S. Food and Drug Administration approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to or whose disease has returned after at least four prior lines (different types) of therapy. Abecma is a cell-based gene therapy approved by the FDA for the treatment of multiple myeloma. Multiple myeloma is an uncommon type of blood cancer in which abnormal plasma cells build up in the bone marrow and form tumors in several bones of the body. This disease keeps the bone marrow from making enough healthy blood cells, which can result in low blood counts.

In January 2021, Biogen Inc., a multinational biotechnology company, announced that it had entered into a collaboration with two U.S.-based biotechnology companies, Atalanta Therapeutics and Genentech, to co-develop RNAi therapeutics for the treatment of neurodegenerative central nervous system (CNS) disorders such as Huntington's disease, Alzheimer's disease, and Parkinson's disease.

Global Cell and Gene Therapy Market: Key Trends

Increasing inorganic growth strategies by the market players

Increasing inorganic growth strategies adopted by the market players for cell and gene therapies is expected to drive the market growth over the forecast period. For instance, in May 2021, Biogen Inc, a U.S.-based multinational biotechnology company, announced topline results from the Phase 2/3 XIRIUS study of cotoretigene toliparvovec (BIIB112), a gene therapy being investigated as a one-time therapy for patients with X-linked retinitis pigmentosa (XLRP). XLRP is a rare, inherited retinal disease that is associated with progressive vision loss as the light-sensing cells of the retina gradually deteriorate. Initial symptoms are difficulty seeing at night, followed by restriction of the field of vision and eventually blindness in most people by the age of 40. Patients living with X-linked retinitis pigmentosa (XLRP) currently have no approved treatments.

Similarly, in March 2021, Biogen, a U.S. based multinational biotechnology company, announced that it had planned for a new gene therapy manufacturing facility at its Research Triangle Park campus in North Carolina, the southeastern region of the U.S. The 175,000-square-foot facility had been designed to scale up the manufacturing of the products that are in Biogen's gene therapy pipeline i.e. BIIB111 (timrepigene emparvovec) to treat the inherited retinal disease choroideremia. The expansion will help Biogen in the development of new revenue source.

Increasing prevalence of various chronic diseases

Increasing prevalence of various chronic diseases such as cancer, multiple myeloma, and others is expected to foster the market growth over the forecast period. For instance, according to the estimates by the GLOBOCAN 2020, around 6,900 new cases of multiple myeloma were diagnosed in South East Asia.

Furthermore, according to the estimates by the GLOBOCAN 2020, around 2,155 new cancer cases were diagnosed in the UAE.

Global Cell and Gene Therapy Market: Restraints

Unfavorable Reimbursement Policies

Kymriah, a CD19-directed genetically modified autologous T cell immunotherapy, has a listed price of US$ 475,000 for patients with acute lymphocytic leukemia and US$ 373,000 for patients with large B-cell lymphoma; and Yescarta (A Cart-T cell therapy )has a listed price of US$ 373,000. Treatment with such high price requires government support in terms of reimbursement policy for the better adoption rate.

However, cell and gene therapy has witnessed multiple setbacks from regulatory authorities in charge of reimbursement policy, which is expected to inhibit the growth of the market. For instance, in December 2020, the National Institute for Health and Care Excellence (NICE), U.K. concluded that Yescarta is a very expensive therapy and hence cannot be considered as a cost-effective use of National Health System (NHS) resources.

To overcome this restrain patients can opt for new reimbursement policies under which the cell and gene therapy is being added/included to avoid high cost and out of the pocket investments

Challenges associated with cell and gene therapy

Challenges associated with cell and gene therapy are enlisted below:

Developers of gene therapies are facing a major challenge of convincing medical settings that they should invest in highly expensive products with the expectation that long-term patient benefits justify the cost
Moreover, revenue of the cell and gene therapies indicated for orphan diseases remain limited due to the small size of patient population, thereby indicating commercial failure as the high cost of developing a treatment makes, it an unappealing prospect for pharmaceutical companies.
Though the therapies are costly, developers claim that the therapies are cost-effective to patients as they permanently receive a cure for the disease. However, it is also possible that future gene therapies targeting more common diseases may be less expensive since manufacturers could recover their research and development costs more efficiently from a larger number of patients.