CMC Expertise Accelerating Biologics Development for 20 Years and Counting

Working with my co-founders, colleagues, and clients on many fascinating projects over the last twenty years has been an honor. From the founding of the firm with former Pfizer colleagues until today, we have specialized in supporting the CMC and regulatory programs...

The Bustling Cell and Gene Therapy CMO Scene

Gene Therapy CMOPharma’s new and exciting era of targeted therapeutics has the potential to be a turning point for modern medicine. The buzz has led to a flurry of investment in the cell and gene therapy space. According to a recent Alliance for Regenerative Medicine report, nearly $20 billion in funding flowed into biopharma companies developing advanced therapeutics last year — shattering the previous investment record of $13.5 billion set in 2018.

With a robust pipeline of potential new cell and gene therapies — more than 3,700 active clinical programs worldwide for cell and gene therapy — the sector is undoubtedly maturing. As more companies come online and more promising new drugs head into late-stage trials, the industry is experiencing a manufacturing bottleneck. In the near future, cell and gene therapies could create treatment options for millions of patients living with difficult and even incurable diseases. But the true potential of these exciting pipelines can’t be realized unless these therapies can be scaled up and manufactured efficiently, consistently, and affordably.

Both small drug developers and large pharmaceutical companies are looking to contract manufacturers for help scaling up and commercializing these new advanced therapeutics. Studies conducted by market analysis company BioPlan Associates have found that approximately 90% of cell and gene therapy developers prefer to use contract manufacturing organizations (CMOs) to commercialize their products. This has led to a pharmaceutical contract manufacturing space wrought with activity as CMOs innovate to address present and future capacity challenges.


Lack of CMO Capacity

Traditional biopharma facilities producing recombinant proteins or monoclonal antibodies are generally designed and constructed similarly, giving CMOs the flexibility to produce different types of drugs using the same facility. However, cell and gene therapies have unique production needs, which means they need uniquely designed facilities. These new therapies require high containment levels, which means CMOs must incorporate additional design elements for segregation, such as fully exhausted biosafety cabinets or segregated air handling units. Add to that, the very complex and manual processing required to manufacture cell and gene therapies necessitates highly trained personnel, and there is currently a  shortage of qualified workers.

BioPlan’s annual survey found that a third of respondents were experiencing significant difficulties hiring cell and/or gene therapy staff. Given these challenges, it should come as no surprise that a limited number of CMOs offer commercial cGMP manufacturing for cell and gene therapies. In fact, according to GlobalData, as of last year, there were only 152 CMOs worldwide with the capabilities to manufacture cell and gene therapies. According to BioPlan, lead times for CMOs to begin cell or gene therapy projects are averaging 18 months — and increasing, This has created a sizeable market opportunity for CMOs who can overcome these obstacles and expand their capacity. These efforts have resulted in several trends within the contract manufacturing space.


CMO Capacity-Boosting Trends


1. M&A

Many CMOs looking to build their manufacturing capacity and capabilities have turned to the tried and true method — acquisitions. The last few years have seen enormous M&A deals within the cell and gene therapy CMO sector. Global CDMO giant Catalent, for example, has been very active in cell and gene therapy-related buys. In May 2019, Catalent plunked down $1.2 billion to snap up Paragon Bioservices, a leading viral vector development and manufacturing partner for gene therapies. Paragon had just opened a new, state-of-the-art commercial manufacturing center in the U.S. and had GMP manufacturing projects underway with more than half of the top 40 leading gene therapy developers worldwide.

Catalent followed this up with a $315 million deal in 2020 to buy MaSTherCell, a provider of cell and gene therapy development and manufacturing services, and through this deal gained facilities in Gosselies, Belgium, including a new fill-finish facility. This year, Catalent purchased Promethera Biosciences’ cell therapy manufacturing subsidiary, Hepatic Cell Therapy Support, gaining an additional facility in Belgium. This was followed by an agreement to acquire RheinCell Therapeutics, a Germany-based developer and manufacturer of human induced pluripotent stem cells. Large deals in the CMO space were not exclusive to Catalent either.

Thermo Fisher Scientific picked up Brammer Bio, a leader in viral vector manufacturing for cell and gene therapies, and its two U.S. facilities for $1.7 billion in 2019. Oxgene, a small UK-based contract research and development company focused on gene therapies, also found itself in need of manufacturing prowess. The company’s solution came in the form of a recent $135 million takeover by Shanghai-based WuXi AppTec, creating a subsidiary, WuXi Advanced Therapies. The deal means Oxgene can rely on manufacturing power from WuXi to scale up its patented systems — designed to produce gene therapy vectors more affordably and at a higher quality — thus facilitating the company’s production of gene therapies.


2. Expansions

Looking to take on more cell and gene therapy clients and larger late-stage proposals, many CMOs are also investing heavily in expansion projects. According to BioInformant, from January 2020 to September 2021, nearly $10 billion was invested into CMO expansions to support cell and gene therapy manufacturing. Tokyo-based CDMO Fujifilm Diosynth Biotechnologies picked up a fourth biologics manufacturing site through an $890 million, all-cash exchange with Biogen in 2019 and then immediately got to work making it bigger, investing another $928 million to double cell culture manufacturing capacity and add drug product manufacturing at the site.

The CDMO’s parent company, Fujifilm Corp., is currently spending more than $2 billion to build a mega-plant for cell culture production in North Carolina, due for completion in 2025. Last November, South Korean CDMO Samsung Biologics broke ground on a $2 billion “Super Plant” that is expected to be accountable for a third of the total global bio-CMO manufacturing capacity once completed. The Super Plant, plus additional construction underway at Samsung’s Incheon hub, will add additional manufacturing capacity and technology platforms to produce cell and gene therapies.


3. New Launches

Arguably the most ambitious solution to the capacity crunch, several new cell and gene therapy-focused CMOs with big potential have recently launched onto the scene. In January 2020, the newly formed Center for Breakthrough Medicines announced its intention to build the world’s largest single-site CDMO dedicated to end-to-end development and manufacturing of cell and gene therapies. The mammoth 680,000-square foot facility will sit inside the 1.6-million square foot Discovery Labs life science campus, an innovation hub based in King of Prussia, Pennsylvania.

Also launched in early 2020, Forge Biologics has a twofold mission — to serve gene therapy developers as a CDMO and to leverage its contract services revenue to fund the development of its own gene therapy pipeline. Forge currently operates 175,000 square feet of modular cGMP manufacturing in Columbus, Ohio. Once its expansion plans are complete, the venture-capital-backed gene therapy startup is poised to be among the top five gene therapy manufacturers globally in terms of capacity. In October 2020,

New Jersey Innovation Institute cut the ribbon on BioCentriq, the first fully cGMP cell and gene therapy contract development and clinical manufacturing center located on a university campus in the U.S. BioCentriq’s newest addition is a fully validated ISO-7 certified clinical manufacturing facility located on the campus of the New Jersey Institute of Technology (NJIT) in Newark, New Jersey. Boasting a somewhat different business model, the CDMO strives to be cost-effective enough to serve startups and small biotechs facing limited resources.


BioTechLogic Can Help You to Navigate the Challenging Landscape

Navigating the exciting waters of a market expected to surge to $15.48 billion by 2025 will require a combined push from pharma manufacturers and CMOs. While contract manufacturers are working on expanding manufacturing capacity for cell and gene therapy as quickly as possible, capacity remains limited, and competition for space remains tight. Additionally, developers looking for contract partners need to vet potential CMO partners and then manage the collaboration throughout the program. BioTechLogic can provide ongoing support and expertise so that developers can work efficiently and effectively with contract partners in order to bring innovative, life-saving treatments to patients who need them.

Contact us to explore how BioTechLogic can help.