Andelyn Biosciences | Top Contract Manufacturing Organizations 2022
Pharma Tech Outlook

Pharma Tech Outlook

Andelyn Biosciences
The Complete Package for Gene Therapy

Andelyn Biosciences: The Complete Package for Gene Therapy

Wade Macedone, COO, Laura Rau Hillock, General Counsel, Eric T. Blair, CCO, Adam Lauber, CFO., Andelyn BiosciencesWade Macedone, COO, Laura Rau Hillock, General Counsel, Eric T. Blair, CCO, Adam Lauber, CFO.
The biopharma industry is tirelessly working to provide high-quality and effective treatment options with new and innovative methods, such as gene therapy. One challenge in a relatively nascent industry is limited resources and expertise. Another challenge is the capacity of the manufacturing operations that restricts the scalability of biopharma operations. The pandemic introduced supply chain challenges which contributed to the lag in scalability, impacting the quantity and speed to market of new drugs.

Andelyn Biosciences overcomes these challenges. With over 20 years of experience in gene therapy research and manufacturing, Andelyn supports clients with diverse capabilities, spanning preclinical process development to small and large-scale GMP clinical and commercial manufacturing, employing both adherent and suspension up to 2000L. Andelyn’s newly added in-house analytics and GMP plasmid manufacturing services supplement its manufacturing services and substantially reduce delivery timelines of gene therapy products to clients. Manufacturing in its two new state-of-the-art facilities with over 250,000 square-foot of space, Andelyn has now expanded its services to include Phase III and commercial manufacturing of gene therapy products, empowering its clients to scale production quickly and efficiently.

On the front-end, we aim to cut downtime, while on the back-end, we simplify long lead assays to provide a one-stop shop and ultimately accelerate speed to market

Andelyn assists and supports its clients through all phases of development and commercialization of therapies, eliminating the need for licensing and technology transfers. “On the front-end, we aim to cut downtime, while on the back end, we simplify long lead assays to provide a one-stop shop and ultimately accelerate speed to market or advance clinical trials. Most CEOs or institutional leaders believe this is the most critical step, and by accelerating production, our clients achieve an important advantage,” says Eric T. Blair, CCO of Andelyn Biosciences.

Andelyn’s journey into the gene therapy realm began at the Research Institute at Nationwide Children’s Hospital (NCH) in Columbus, Ohio, one of the largest pediatric hospitals and research institutes in the United States. NCH’s physician-scientists collaborated with hospital and research leadership, leveraging decades of experience in the pharma space to create a small GMP facility to support pediatric research. This facility produced viral vectors for pivotal gene therapy clinical trials, accelerating NCH’s gene therapy program. “Andelyn” is named after two pioneering patients who participated in pivotal clinical trials at NCH—Andrew and Evelyn. After much success and clear industry demand, Andelyn was spun out of NCH in September of 2020 as a for-profit contract development and manufacturing company. These two pioneers continue to inspire Andelyn’s employees and serve as the foundation for Andelyn’s mission, to deliver lifesaving therapies to patients by Pioneering Solutions to Bring More Treatments to More Patients. The passion behind the work is a key reason that Andelyn, alone and as part of NCH, has been able to support over76 INDs and over 452 GMP batches demonstrating the leadership, experience, and trust only a patient-focused organization can achieve in such a short period of time. Now with 250,000 square-feet of high-tech space, Andelyn is ready to scale its impact to satisfy demand from the Gene Therapy community.

End-to-End Gene Therapy

As a full-spectrum gene therapy partner, Andelyn brings Pioneering Solutions that Turn Hope into Reality by expediting the development of leading-edge gene therapies. Andelyn’s clients include big pharma, biopharma, biotech companies, and smaller gene therapy development companies.

It also includes a focus on the researchers and clinicians at academic research institutions, private foundations, and government organizations that provide a high degree of innovation to new therapies and modalities. Andelyn’s engages each client with a thorough assessment of the client’s goals, indications, and timelines and then develops a plan to address their needs, from materials sourcing to clinical and commercial production to approvals. Andelyn also advises less experienced clients on how best to use the gene therapy platforms and materials available to achieve the desired yields. Experienced clients benefit from Andelyn’s experience and knowledge to help accelerate the development curve. These flexible models of service and knowledge exchange create trust between organizations and ultimately deliver the best combination of materials and platforms for each client program. The highest yields translate into the production cost, resulting in more patients being treated.

Andelyn deploys an experienced project management team for each client that enables timely intake and management of each client program with the right expert support. Proactive discussions during the entire product lifecycle and regarding its commercial validation save time and effort for clients. Andelyn’s cutting-edge technologies enable innovative companies and institutions to advance in the field of gene therapies while offering patients life-altering therapies.

With a broad range of viral vector manufacturing platforms and solutions, including AAV adherent and suspension systems, plasmid manufacturing, optimized cell lines, and analytical testing, Andelyn supports clients through every step of cGMP manufacturing. Andelyn is also unique in that its process development and plasmid analysis services are available across the variety of serotypes required for gene therapy production to provide the best probability and success of a commercially viable gene therapy product. In addition, Andelyn has dedicated production suites for manufacturing research-grade and toxicology-grade viral vectors that support preclinical studies.

Regulatory compliance is key to Andelyn and is built into its operations and DNA. Andelyn mitigates risk by implementing well-established processes developed over decades that utilize the latest manufacturing optimization tools to maximize efficiency and accelerate timelines. Through the process, Andelyn is compliant with global regulatory bodies and has supported more than 76 investigational new drug (IND) applications for viral vector-based gene therapy products around the world. As a key player in the community to support Gene Therapy success, Andelyn works closely with the regulatory bodies globally to share appropriate feedback and ensure a safe, efficacious, and effective production of Gene Therapies.


Reliably Expert, Purposely Driven® Experience and Problem Solving is the DNA of Andelyn

“At its core, Andelyn brings a problem-solving approach driven by decades of experience and knowledge,” says Adam Lauber, CFO of Andelyn. Its highly qualified team brings rich expertise to the table, providing innovative solutions to clients’ challenges. Our NCH roots have provided us with significant experience in analytics, so we have capitalized on this advantage by bringing all key analytics in-house to remove production delays caused by release-testing performed at third-party labs.”

  • At its core, Andelyn brings a problem-solving approach driven by decades of experience and knowledge

Another area presenting obstacles to accelerating Gene Therapy-productions are plasmid production delays. In response, Andelyn invested in bringing GMP plasmid production in-house to match its current research and tox-grade plasmid production offerings. Andelyn believes that this starting material is so critical that they launched their programs to use single-use consumables for all productions in for both toxicology and GMP runs. In addition, all GMP production is performed in a clean room, regardless of the clinical phase it is supporting. These in-house analytical and plasmid capabilities will accelerate timelines and ensure quality for Andelyn’s clients. Through control and support of the entire process, Andelyn reduces its clients’ time to production creating a measurable advantage to accelerate program through the by greater than 60% percent.
Building Partnerships

Rather than looking at short-term goals, the company aims to develop relationships and strategic partnerships with its clients. Andelyn has been the catalyst in over 76 INDs, with the successful completion of more than 452 cGMP clinical batches and over 2,000 research-grade batches. Andelyn’s deep-rooted experience gives the company a leading position in the market, promises outstanding results for its clients, and builds on the trust first developed over 20 years ago with its first research and development partners while part of NCH.

“This experienced Andelyn team helped write the book on Gene Therapy manufacturing. What we have accomplished in 20 years operating out of such a small facility of only 9,000 square feet at NCH is amazing. Our team made history when we supported the clinical trials of the very first FDA-approved gene therapy, Zolgensma®,” said Andelyn COO Wade Macedone. “With the investment by NCH to spin out Andelyn as a 100 percent CDMO, we now have the best of both worlds and room to grow. However, we will always maintain our focus on the patient, researchers, and clinicians as we expand operations in our 250,000 square feet of state-of-the-art facilities. This places Andelyn among the largest CDMOs in the world for gene therapy and provides us with the capacity to partner with our clients from concept to commercialization.”

“As a 100 percent CDMO, we will never compete with our clients – we are here to support and facilitate quality products and services to yield the very best results to treat patients faster,” Blair added. “Andelyn’s regulatory support and supply chain vertical integration help accelerate the development and manufacturing of innovative therapies to bring more treatments to more patients.”

There have been several instances where clients came to Andelyn unhappy with the outcomes from other CDMOs and have achieved success with Andelyn. Andelyn helps its clients turn around programs, and in one case. In another example, a biotech client reached out to Andelyn to bring a product into phase I quickly, in seemingly unrealistic timeframes. Here, Andelyn leveraged its manufacturing and regulatory experience to support the client in their IND and delivered the product within the timeframe. Additionally, the client shared that it was able to get it approved by the FDA without any comments or findings, a rare achievement. Andelyn’s expertise and deep understanding of FDA requirements allowed the company to support a successful IND start clinical trials in a crucial program.

At the end of the day, Andelyn’s goal is to partner and provide clients with the highest yielding and quality product on the timeline needed to reach patients. Andelyn’s success in achieving this goal is reflected in a positive outcome achieved by a large biotech client resulting in an increase in yield by 45 percent after Andelyn identified an issue in client’s process and optimizing a critical assay without changing reagents. Andelyn worked with the client’s existing reagents, resulting in higher yields and allowing patients to benefit more from the same manufacturing lot.

As Andelyn unveils its newest 200,000 square foot facility and headquarters, it is set to boost its current production capacity by a multiple of 25, offering immense scalability to clients with over 14 upstream GMP manufacturing suites. Andelyn is also establishing itself as a leader with a Digital CDMO service that will leverage next-generation tools to provide manufacturing data as a tool for clients as well as real-time person-in-plant video for remote access to productions. This revolutionary service links manufacturing data to patient insights. With Andelyn, clients gain access to innovative manufacturing capabilities and develop therapies for rare to common diseases cost-effectively and with the highest quality and expertise. By providing clients with reliable and responsive manufacturing services, and with this expanded capacity, Andelyn will continue to aid the advancement of gene therapies through the clinic and to market at an unprecedented pace. On a daily basis, Andelyn differentiates itself as an expert, trusted, and flexible partner – mission-driven to accelerate development and manufacturing of innovative therapies to Bring More Treatments to More Patients™ through Pioneering Solutions that Bring Hope Into Reality™.

Top 10 Contract Manufacturing Organizations - 2022

Company
Andelyn Biosciences

Management
Wade Macedone, COO, Laura Rau Hillock, General Counsel, Eric T. Blair, CCO, Adam Lauber, CFO.

Description
Andelyn Biosciences supports clients as a 100 percent CDMO in Gene Therapy with diverse capabilities spanning preclinical R&D, small to large scale GMP manufacturing up to 2000L, Analytical Development and testing, and plasmid manufacturing services.