Five Companies That Are Powering The Cell and Gene Therapy Revolution
Several Maryland companies are making a global impact in the field of cell and gene therapy
July 16, 2019
The promise of cell and gene therapies to cure unmet medical needs is finally starting to come through as life-saving new medicines are now hitting the market every year. The global market for gene therapies was estimated at $2.3 billion in 2018 and it is expected to hit a compound annual growth rate of 49.9% to reach $17.4 billion in 2023.
The 2017 FDA landmark approval of Novartis’ KYMRIAH to treat a rare form of childhood blindness marked the beginning of a revolution in the cell and gene therapy field. Since then we have seen that the advances in the field are finally producing results and several more products have received FDA approval. Recent notable approvals include Spark Therapeutics’ LUXTURNA, Kite Pharma’s YESCARTA, and the most recent approval of ZOLGENSMA developed by AveXis (a Novartis company).
While these big therapeutic companies are taking the top headlines, there is a whole ecosystem of innovative biotech and life science technology companies behind them that are developing the tools, technologies, and infrastructure that is propelling the industry forward.
We have identified five companies that stand out for their unique technologies and solutions that are responsible for driving progress in the cell and gene therapy field and helping their partners bring their products through the clinic and to the patients that need them.
#1. Paragon Gene Therapy
Paragon Gene Therapy has dominated the cell and gene therapy headlines this year culminating in the $1.2B acquisition by Catalent, a world leader in contract development and manufacturing (CDMO) services. The acquisition came just after Paragon announced the grand opening of their new 150,000 sq/ft cGMP manufacturing suites which expanded their capacity to provide late-stage gene therapy development and manufacturing solutions.
Paragon Gene Therapy is a Baltimore, Maryland-based CDMO that has grown rapidly over the past five years on the foundation of their unparalleled expertise in gene therapy manufacturing for clinical and commercial therapeutics. The development and manufacturing processes for gene therapies are both highly complex and evolving, which is why many therapeutics companies are choosing to outsource production to specialists like Paragon. Paragon has projects underway with 20 of the worlds top 30 industry leaders, including; Sarepta Therapeutics, IVERIC bio, Inc. and Amicus Therapeutics, Inc. which were all announced in the past 60 days.
The latest strategic client acquisition may be their most high profile to date. AveXis, a Novartis Company, announced on July 16th that they selected Catalent’s Paragon Gene Therapy as an additional commercial manufacturing site for its spinal muscular atrophy (SMA) gene therapy Zolgensma®. Zolgensma received the FDA’s first approval for an SMA gene therapy earlier in May of this year and has received a lot of attention for its list price of $2.1 million. The long-term strategic agreement with Paragon also includes process development support for the clinical supply of additional viral therapies in AveXis pipeline.
Paragon has propelled itself into a leading position within the gene therapy market through its keen leadership and strategic capital investments. Starting with their investment in building out several new cGMP facilities to the most recent acquisition of two of Novavax’s manufacturing facilities and more than 100 employees that will immediately extend their production capabilities. Coupled with the now global reach of their parent company Catalent, Paragon will become an end-to-end, pre-clinical through Commercial, CDMO with a strong position to dominate the gene therapy market.
“Paragon has set the standard for other CDMO’s around the world in its pursuit of biomanufacturing excellence,” stated Marty Rosendale, CEO of Maryland Tech Council at the Grand Opening celebration for one of Paragon’s new facilities. “The bar has been raised by the company whose very name means the standard and center of excellence.”
MaxCyte stands out as a unique biotech company that is helping to unlock the full potential of cell and gene therapies by fueling the progress of its many partners and collaborators. Part of what makes MaxCyte unique is that the company is not only a leading life sciences instrumentation company for cellular engineering, but it is also advancing its own portfolio of clinical-stage cell-based therapies.
Founded in 1999, MaxCyte’s core technology is a proprietary, advanced cell engineering platform that is enabling leading companies like Kite Pharma, a Gilead Company, and CRISPR Therapeutics to advance their cell-based medicine to patients with high unmet medical needs.
To benchmark the level of impact MaxCyte is making in the field, the company now has more than 70 partnered program licenses in cell therapy with more than 35 licensed for clinical use, including four announced commercial licenses covering potentially more than 30 products. One such program that was announced in March of this year is a multi-drug clinical and commercial agreement with Kite, a Gilead Company, who will use MaxCyte’s Flow Electroporation® Technology to enable non-viral cell engineering for the development of multiple CAR-T drug candidates for up to 10 targets.
Earlier this year the company launched the new ExPERT™ technology platform. This family of instruments represents the next generation of the industry’s leading, clinically validated Flow Electroporation® Technology for complex cellular engineering.
“Creating cellular editing platforms standardized on a single, scalable, high-performance technology can assist the industry in accelerating timelines, reducing costs and achieving milestones critical to the translation of this promising new generation of cellular therapies,” shared Brad Calvin, Executive Vice President of Global Commercial Operations for MaxCyte.
CRB is one of the largest design, engineering, construction and consulting firms for the life science industry, and they have unique expertise in the cell and gene therapy space. Since the nacent cell and gene therapy field is evolving so rapidly, for companies to be successful they need to understand and reimagine almost everything in their operation—from supply chain and logistics to workflow, manufacturing, quality, safety, and labor. Each of these components impacts facility design and engineering. It’s such a new industry that many processes haven’t even been developed, making it all that more challenging for companies to navigate their growth. This is where CRB comes in with the expertise to help companies navigate a sea of challenges during their relatively uncharted journey.
One component that CRB brings to the table that sets them apart from other firms is a team of Process Architects, like Grace Linton, RA, AIA, LEED AP BD+C. Worldwide, process architects are a rare breed. Very few companies have one process architect, let alone a team of them. With specialized expertise as licensed architects who are well versed in building codes, building systems and materials, and architectural design, and also cross-trained with an in-depth understanding of process, operational and cGMP regulatory requirements and knowledge of equipment and technology.
The common challenge that most cell and gene therapy companies face is that many of their processes are still evolving or undefined, and most development and manufacturing is done using manual, open processing due to lack of automation options. This makes everything more complicated for regulations and for facility design and operations and is the precise type of scenario that CRB’s process architects are trained to develop solutions for. The early involvement of a process architect can also help companies properly plan the facility for scaling out, and future proof design strategies. Accounting for these scenarios early in the design process for cell and gene therapy production facilities can save the company from expensive challenges later on.
Secondly, CRB is an industry leader in a new integrated project delivery methodology for facility design and construction. In a field where time and money matter in order to bring new therapies to patients cheaper and faster, CRB is becoming a partner of choice for many cell and gene therapy companies. The basics of this framework are somewhat equivalent to Agile Development for software. The methodology promotes iterative design, engineering and construction throughout the life-cycle of the project, close collaboration between the design and construction teams, subcontractors and client-side, constant communication, and tightly-knit teams focused on a single goal – delivering exceptional results for the client.
CRB applied this new methodology to help Paragon build out its new cGMP gene therapy manufacturing facility that opened earlier this year. “It was this approach that made it possible to deliver 75,000 sq feet of functional space in a record-worthy 15 months after kick-off, helping to put the company in a position to confidently bring on new client programs and grow their pipeline.” shared Matt Khair, CRB’s Senior Associate at the grand opening event for Paragon’s new facility.
#4. Lab Owl
Lab Owl™, operating out of Rockville, Maryland, provides a fully customizable and scalable platform for bioreactor controls. First introduced to the bioscience market in 2017, the technology behind Lab Owl has evolved through many years of custom automation solutions for top biopharma companies. Lab Owl is a spinoff from New Jersey-based Automated Control Concepts Inc. who has been helping biopharma companies automate complex manufacturing processes for more than three decades.
Lab Owl™ was built to bring flexibility and modern automation technology into development labs and small-scale production facilities, providing a welcomed solution to many cell and gene therapy companies.
Cell and gene therapies are produced in much smaller batches and require a different approach than that of more traditional biomanufacturing processes that are designed to produce large batches. This customized, targeted approach creates a unique set of challenges that Lab Owl is especially suited to solve.
Cell and gene therapy production challenges are rooted in the need to scale-out manufacturing, or develop multiple batches simultaneously that are personalized for each new patient, and the need to manage a small-scale open processing environment. The result is a company operating an uber flexible development facility and managing multiple, small, benchtop bioreactors, single-use manufacturing strategies, cutting-edge equipment and a retail-like supply chain.
Lab Owl helps cell and gene therapy organizations get control of and improve lab performance, even in an industry that’s still trying to discover the best path forward. Their platform is technology agnostic, extremely flexible, modular and scalable which helps to solve many of the challenges these companies face as they scale up and move their products to and through the clinic.
These uniquely tuned platform features, combined with their keen ability to understand and develop solutions for each customer’s unique methodologies, a skill that has been honed in their 30+ years under ACC, has made them a go-to partner for companies in this diverse and growing field. We expect to see Lab Owl continue to grow in parallel to the rapidly expanding cell and gene therapy field.
#5. Lentigen Technology, a Miltenyi Botec Company
Lentigen was founded in 2006 and has grown to become an industry-leading provider for the design, construction and production of lentiviral vectors for pre-clinical and clinical applications. More than a third of ex vivo modified gene therapy clinical trials use a lentiviral delivery method, and more than 100 clinical trials have now conducted or are in progress using lentiviral vectors, with significant success in LV-modified CAR-T cells and also LV-modified stem cells.
Lentigen was founded in a UMBC incubator by Boro Dropulić, who is now Lentigen’s Chief Science Officer and General Manager, and later relocated to Montgomery County, Maryland. The company was purchased by Miltenyi in 2014, and is now in the process of relocating to a much larger 150,000 sq ft manufacturing facility, the former Qiagen building in Gaithersburg, MD, to meet the demand for their lentiviral solutions.
With such a sizable market, there is a growing need among therapeutic companies for a partner to help them harness the power of lentiviral gene delivery and expression technologies and bring their therapies through the clinic.
What makes Lentigen stand out, even more, is that as a subsidiary of Miltenyi Biotec GmbH, a pioneer in developing innovative workflows, tools, and technologies for clinical cell and gene therapy applications, Lentigen can help facilitate end-to-end support for your ex vivo cell processing with sophisticated automated workflows and optimized reagents for cell manufacturing.
The fledgling cell and gene therapy space is presenting many new challenges for this new wave of therapeutic companies. Those challenges range from automation and production to logistics, and many are debating the path forward. One such debate is whether a centralized production model or a decentralized (point-of-care) model is best, especially for delivering autologous therapeutics to patients. Myltenyi is betting on the decentralized model.
Their CliniMACS Prodigy®, an all in one automated T cell expansion and purification system, has the potential to tip the scale for the “point-of-care” model of cell manufacturing/ delivery. If successful, Prodigy® could bring an end to complicated cleanroom facilities and simplify the whole manufacturing process right on sight at the point of care. There is a lot of progress needed to make this a reality, but Miltenyi is at the forefront of making this vision a reality.
With Lentigen’s growing position as a leader in the lentiviral production market and unique access to Miltenyi’s global client footprint, they are positioned to continue to help fuel the cell and gene therapy field and make a substantial impact on the future of medicine.
There are many more notable companies that deserve recognition for their contributions to helping advance the field with innovative solutions to the many ongoing challenges that cell and gene therapy companies face. In Maryland, companies like RoosterBio, Precigen, Thermo Fisher are worth mentioning. There are also several companies with platform technology solutions such that are poised to make a significant impact. Companies such as REGENXBIO, Immunomic Therapeutics and American Gene Technologies are all developing their own pipelines while also advancing a platform that others can license to bring their therapeutics to market.
The one thing we know for sure is that this industry is still young and there are some exciting times ahead because of the work being done by innovators around the world.