Gathering at the Mesa to Showcase Opportunities and Challenges in Cell and Gene Therapy

Gathering at the Mesa to Showcase Opportunities and Challenges in Cell and Gene Therapy

The 2024 Cell & Gene Meeting on the Mesa has changed its venue this year, taking place from Oct. 7–9 in Phoenix, after previously being held in Carlsbad, California. The industry remains entangled in persistent regulatory, manufacturing, and commercialization hurdles despite this venue shift, as the field approaches a critical turning point.

BioSpace will cover this three-day gathering, which is expected to attract over 2,000 participants keen on updates from influential industry stakeholders regarding advancements and challenges in cell and gene therapies (CGTs).

Notably, the sector faces significant investment difficulties, as highlighted by the Alliance for Regenerative Medicine (ARM), a DC-based advocacy group hosting the Meeting on the Mesa.

Stephen Majors, ARM’s global head of communications, shared with BioSpace that the capital markets have presented considerable challenges for CGT over recent years. He noted that numerous companies are now seeking smaller amounts of funding.

“Inflation concerns have particularly impacted gene therapy companies, which are usually farthest from commercialization,” Majors remarked. “We maintain hope that as interest rates decrease and inflation outlook improves, investment will increase in the sector,” he added.

Manufacturing, Commercialization Challenges

Majors further elaborated on the manufacturing difficulties plaguing the industry due to a lack of standardization, where scaling up production often complicates regulatory approvals and commercialization efforts.

“Manufacturing hurdles still exist, particularly regarding the ability to produce therapies at the required scale,” Majors noted.

A Government Accountability Office report from last year concluded that while regenerative medicine, including gene and cell therapies, shows great promise, lack of universal standards and guidelines hinders their development and implementation.

At the 2024 American Society of Gene & Cell Therapy conference, Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), provided a candid overview regarding the prospects and challenges within the CGT realm. He noted that despite the promising future for gene therapies—like those approved for sickle cell disease—several significant issues persist, including manufacturing complications and varying global regulatory landscapes.

Recently, infusions of sickle cell therapies from Vertex-CRISPR Therapeutics and bluebird bio commenced, igniting optimism among both patients and these companies striving to catalyze revenue streams.

Majors commented on the anticipated gradual uptake of these therapies, highlighting the extensive time needed for identifying eligible patients and consulting with physicians about the appropriateness of treatments.

At this year’s Meeting on the Mesa, a session focused on the commercialization of cell and gene therapies promises to generate interest, especially given the recent product rollouts, which may yield valuable insights.

In a returning conference feature, ARM CEO Tim Hunt will lead a session centered on ethical considerations in CGT. This broad-based discussion will encompass various topics, such as the pricing and value of therapies, global access logistics, and addressing ultra-rare conditions that might not fit commercial models effectively.

As reported by market analysis firm Nova One Advisor, the global CGT market was valued at over .1 billion in 2023, with projections indicating a rise to approximately .3 billion by 2033.

While the development journey for CGT encompasses numerous various stages, the firm emphasizes that enabling patient access while managing supply chains and adhering to complex regulatory mandates proves more burdensome for CGTs than for traditional pharmaceuticals.

Regulatory Developments Take Center Stage

As part of the regulatory discussions at Meeting on the Mesa, Nicole Verdun, director of the FDA’s Office of Therapeutic Products at CBER, will present updates on the FDA’s perspective on cell and gene therapies, including staffing to handle submissions and insights into accelerated approvals.

Scott Gottlieb, former FDA commissioner, will also engage in a fireside chat at the conference. In 2019, Gottlieb speculated that the FDA might approve between 10 to 20 CGT products annually by 2025. His forthcoming book, The Miracle Century: Making Sense of the Cell Therapy Revolution, aims to address key issues crucial for the broad adoption of these treatments.

Majors states that Gottlieb will cover the current state of the field and future expectations.

“We have observed little difference in approval rates historically between the U.S. and Europe,” Majors noted, adding that there’s been recent momentum in U.S. approvals.

In its yearly Cell & Gene State of the Industry Briefing, ARM projected 17 regulatory approvals across the U.S. and EU this year. However, that estimate was revised downward to a forecast of 14, which includes eight anticipated approvals in the U.S.—notably, Orchard Therapeutics’ first FDA approval in March for an autologous gene therapy addressing the rare childhood disorder, metachromatic leukodystrophy, and Pfizer’s approval in April for its hemophilia B gene therapy, Beqvez.

“Last year marked a significant breakthrough for gene therapy, while 2023 has showcased considerable advancements in cell therapies,” Majors remarked. There have been impressive fundraising surges from various cell therapy firms, alongside notable progress in treating autoimmune disorders.

Earlier this year, the FDA approved Iovance Biotherapeutics’ lifileucel as the inaugural one-time cell therapy for solid tumors, alongside two new CAR T therapies for earlier treatment of multiple myeloma also receiving approval in April. August saw Adaptimmune Therapeutics’ Tecelra achieve FDA approval as the first engineered cell therapy for solid tumors.

Majors highlights the significance of Autolus Therapeutics’ upcoming FDA application for the next-gen CAR T therapy obe-cel, with its PDUFA target action date set for Nov. 16, aimed to outpace existing CD19 CAR T therapies in terms of clinical effectiveness and safety.

This past January, the FDA called for a class-wide boxed warning for all commercial BCMA- and CD19-directed CAR T cell therapies, stemming from risks linked to T cell malignancies. Majors mentioned that although a correlation exists between these therapies and secondary cancers, the advantages they offer still outweigh the associated risks, proving critical for physicians managing severe cancer cases.