Taking an Industry Pulse at Meeting on the Mesa

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In just two weeks, many in the gene and cell therapy field (including me) will be heading to the Alliance for Regenerative Medicine’s 2024 Meeting on the Mesa in Phoenix. I’m looking forward to connecting with peers and enjoying the warmth of Arizona in October. More importantly, though, the annual event provides an opportunity for me to take a pulse on the advanced therapies industry.

I’ll be paying close attention to sessions with key United States Food and Drug Administration (FDA) experts Scott Gottlieb, M.D. and Nicole Verdun, M.D., as well as Yoshiaki Maruyama, Ph.D. of Japan’s Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA). Regulators have made it clear that they aim to address key challenges to the development of gene and cell therapies, especially for rare diseases, and they want to work with developers in this effort. The Advanced Medicine Partners team has seen firsthand just how critical a robust CMC package is for getting new gene and cell therapies on the market and in the hands of patients. All too often, developers get slowed or even halted by immature manufacturing processes that produce drug product with unacceptable levels of process and product-related impurities. Choosing a CDMO with experience can ensure a drug substance of optimal quality is produced. I’m curious to hear what Drs. Gottlieb, Verdun and Maruyama will add on this topic. Additionally, I believe we’ll hear from them that increasing alignment between FDA and ex-US agencies, like PDMA, should be prioritized to increase the efficiency with which advanced therapies are approved.

Outside of regulatory discussions, I’m eager to spend time interacting with developers during my time in Phoenix. Learning about novel capsid programs being pursued informs how I (along with the full Advanced Medicine Partners team) can best support potential partners. We take pride in our approach of using the proven “spine” of our manufacturing process along with bespoke elements tailored exactly to the needs of a specific program. Personally, finding unique and creative manufacturing solutions without ever compromising on quality is one of the best parts of my job. I also like focusing on helping newer AAV-based companies advance their programs rapidly and cost-effectively, again maintaining the highest quality. Advanced Medicine Partners has developed a robust and reproducible manufacturing process that is commercially viable and highly productive for AAVs, including best-in-class analytics and characterization. Our innovative AAV process is one of the most productive in the industry and consistently generates >95% functional full capsids.

If you’ll be in Phoenix, stop by the Advanced Medicine Partners booth (FLW Building, Table F21) and join us for our CEO Andrew Knudten’s presentation on Tuesday, October 8 at 10:45 a.m. MST in FLW Ballroom G. I hope to meet you in person in just a few weeks, but if you aren’t going to be at the Meeting on the Mesa, you can reach us at partnership@ampgtx.com.

Joe Balleydier
Senior Vice President, R&D and Innovation

Three Questions with CEO Andrew Knudten

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  1. What excites you about the gene and cell therapy industry right now?
    We are fortunate to be part of an extremely exciting time for gene and cell therapy product development. Advances in technology, the sheer number of programs in development as well as recent product approvals all point toward continued progression. One thing I’m particularly excited about is that regulators are supportive of the rapid growth we are seeing in the gene and cell therapy space. The U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research has made it clear that they aim to address key challenges to the development of gene and cell therapies, especially for rare disorders, and they want to work with sponsors to advance medicines for patients. This is great news, but I think we as an industry play a key role in that effort. Frankly, we need to make the jobs of regulators easier, especially in the area of manufacturing and analytical testing.
  2. That’s not something you hear very often. What exactly do you mean when you say the industry should make the jobs of regulators easier?
    Industry and regulators are sometimes framed in opposition, but we are on the same side. We all want to see safe and effective treatments reach the patients and families who need them. As an industry, our approach to manufacturing, analytical method development, and QC testing has not fully enabled the development of therapeutics regulators want to see and patients are desperately seeking. We need to evolve as an industry and begin delivering the quality of product that patients deserve and regulators expect. In the past, it may have been that technology was still catching up, but now we have access to the technology we need. It’s not just technology, though. We need to have people with the breadth of experience and expertise needed to harness the technology. Simply put, more time and energy needs to be invested in CMC product development so that CMC is not a major topic during regulatory interactions. How great would it be for regulatory interactions to focus exclusively on preclinical data review and clinical trial design instead of CMC-related topics? Choosing a CDMO with experience can help achieve this goal for complex gene and cell therapy medicines.
  3. What are some of the common CMC challenges that come up in regulatory discussions? Are they solvable?
    One issue that has been widely scrutinized is product purity or rather a product’s impurity profile. Immature manufacturing processes may produce drug product that has unacceptable levels of process and product-related impurities. Regulators assess a product’s impurity profile as part of their quality and safety assessments. Another challenge biopharmaceutical companies may come up against is bridging between an early manufacturing process and a commercially-viable manufacturing process. Ideally, sponsors should develop a commercially-viable manufacturing process at the onset of a program. Financial and strategic considerations, though, do not always allow for this. Some sponsors may also find themselves in a situation where they’ve inherited a product that has promising clinical data but was supplied with an immature manufacturing process. This might seem provocative, but sponsors should not be afraid of comparability or bridging studies! Recently, regulators have reinforced that they are very open to modifying a gene or cell therapy manufacturing process if the sponsor can confirm the dose, demonstrate process control and its impact on critical quality attributes, and prove analytically the product produced via a new manufacturing process is more pure than the previous iteration. An experienced CDMO like Advanced Medicine Partners can help a sponsor develop a prospective comparability plan that will be looked on favorably by the regulators. We do this through employing advanced analytics to clearly quantify attributes of product made through each manufacturing process. This allows us to ensure product quality is better through the new manufacturing process and that we are delivering an accurate and precise dose. Sponsors should not be satisfied with a sub-optimal manufacturing process. Patients deserve better.

If you’re curious to know how the Advanced Medicine Partners team can work with you to ensure a robust and proactive CMC approach, reach out to us at partnership@ampgtx.com.

Advanced Medicine Partners Executes Additional $32 Million Financing Agreement led by Deerfield Management, Appoints Two New Board Members

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  • Financing will bring total raised from Deerfield Management, ARCH Venture Partners, and other investors to $60 million
  • Ray Kaczmarek and Felix Hsu appointed to Board of Directors, expanding the company’s contract manufacturing expertise
  • Kaczmarek to serve as Executive Chair of the Board

DURHAM, North Carolina – June 5, 2024 – Advanced Medicine Partners, an organization that delivers process development, manufacturing, analytical development and testing for advanced therapy products with a specialty in viral vectors for gene and cell therapies, today announced a new financing agreement led by Deerfield Management, with additional support from ARCH Venture Partners and other investors.  Upon completion of this latest financing round, the total direct investment in Advanced Medicine Partners since its separation from Jaguar Gene Therapy will tally up to $60 million.

Advanced Medicine Partners also announced the appointments of Ray Kaczmarek and Felix Hsu to the company’s Board of Directors. Mr. Kaczmarek will serve as Executive Chairman. Existing members of the Board include Jonathan Leff, Partner at Deerfield Management Company and Chairman of the Deerfield Institute; Elise Wang, Partner and Head of Private Equity Capital Markets at Deerfield Management Company; David Greenwald, Ph.D., Vice President of Business Development at Deerfield Management Company; and Andrew Knudten, CEO of Advanced Medicine Partners.

“The gene and cell therapy space is at an exciting inflection point and in need of new manufacturing solutions that can efficiently and reliably bring promising precision therapies to patients at scale,” said Jonathan Leff. “We are pleased to support Advanced Medicine Partners and its newest board members with additional capital, helping the company realize its mission to address capacity constraints across the industry while enhancing quality in the design and manufacturing of this therapeutic class.”

“Ray Kaczmarek brings deep knowledge of CDMO operations and biopharmaceutical manufacturing to Advanced Medicine Partners, and his leadership philosophy aligns well with our distinct company culture,” said Mr. Knudten. “Our board will also greatly benefit from Felix Hsu’s proven expertise in building out a successful CDMO business. Both of these experienced leaders will be invaluable resources as we continue to accelerate our company’s growth and deliver best-in-class manufacturing and analytics to our partners.”

Mr. Kaczmarek is a seasoned biopharmaceutical leader with more than 20 years of experience in manufacturing and development. Previously, he served as Chief Executive Officer of Genezen Laboratories, Senior Vice President of GMP Operations for TriLink Biotechnologies, President of Nitto Avecia Pharma Services, and Vice President of Commercial Manufacturing and Supply Operations for Pacira Biosciences, Inc. He is also a proud veteran of the United States Army.

“I’m excited to join Advanced Medicine Partners and contribute to the company’s strategic planning as well as support the strong leadership team,” said Mr. Kaczmarek. “I’m confident Advanced Medicine Partners has the expertise, track record and best-in-class platform necessary to be the gold standard in manufacturing and analytics for gene and cell therapies in the eyes of regulators, biopharmaceutical companies and investors.”

Mr. Hsu is a C-suite executive whose career spans more than 30 years of focus on building businesses and delivering revenue and profit growth. He previously served as Senior Vice President of WuXi AppTec and President of WuXi AppTec Advanced Therapies, Senior Vice President of WuXi AppTec and President of the U.S. Business Unit, and Vice President of Supply Chain Integration at Medtronic. He is also a co-founder, CEO, and Chairman of the Board of the Brain Cancer Research Alliance, which raises awareness and funding for brain cancer research to enable new treatment options.

“I’m pleased to join the Board of Directors for Advanced Medicine Partners during this critical building phase of the company,” said Mr. Hsu. “I look forward to working with other board members and the executive leadership team to ensure the company produces consistent growth and profitability.”

ABOUT ADVANCED MEDICINE PARTNERS
Advanced Medicine Partners, LLC is driven to accelerate the development of innovative advanced medicines to enable partnering companies to make a positive difference for the patients they serve. With one of the most experienced genetic medicines teams in the industry – at all levels, from bench to boardroom – the company uses its expertise to produce best-in-class advanced therapy products and analytics with a specialty in viral vectors for gene and cell therapies. Advanced Medicine Partners’ exclusive manufacturing platform consistently produces yields that are among the highest in the industry and purity profiles that continue to push the boundaries of what’s possible. For more information, please visit www.ampgtx.com and follow Advanced Medicine Partners on LinkedIn.

ABOUT DEERFIELD MANAGEMENT

Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. The Firm works across the healthcare ecosystem to connect people, capital, ideas and technology in bold, collaborative and inclusive ways. For more information, please visit www.deerfield.com.

There’s No Substitute for Deep Analytical Experience

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Unparalleled Analytics for Gene and Cell Therapies
At the heart of pioneering safe and effective gene and cell therapies lies a crucial component: advanced analytics. Advanced Medicine Partners prides itself on deploying cutting-edge analytical techniques to fully characterize and optimize a manufacturing process from the earliest stages. We understand that manufacturing a therapeutic product with an excellent impurity profile contributes to improved safety and patient outcomes, and we aim to leverage our best-in-class analytics (and manufacturing platform) to raise the bar on what excellence means when it comes to producing cell and gene therapies.

Excellence in Analytical Development and Quality Control
Located in Durham, Advanced Medicine Partners’ center of excellence for analytical development and quality control testing showcases our commitment to innovation. Our team’s development of over 50 internal, off-the-shelf methods and 19 external methods accelerates product characterization and reduces timelines.

Specializing in analytical chemistry, molecular biology, microbiology, and cell-based assays, we have made significant strides in functional potency assays for AAV products, demonstrating our broad and deep analytical capabilities.

Efficient and Accurate Turnaround for Faster Advancements
Efficiency and accuracy is key in the fast-paced world of therapy development. With a typical turnaround time of just 10 days for internal testing, we are dedicated to facilitating rapid progress from laboratory discoveries to clinical applications.

Proven Track Record in Supporting Commercial Success
The bottom line: there is no substitute for deep analytical experience. Advanced Medicine Partners’ expertise is not just theoretical but proven through our active role in developing and qualifying analytical methods for multiple commercial products, including three globally approved gene therapies. This experience underscores the irreplaceable value of deep analytical knowledge in achieving commercial and therapeutic success. We want the same for you.

We’re eager to extend our analytical expertise to your projects, offering the same level of support, and scientific rigor that has led to our successes. To explore how Advanced Medicine Partners can use our analytics to assist in elevating your cell and gene therapy products, reach out to us at partnership@ampgtx.com.

Advanced Medicine Partners Announces New Collaboration

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Advanced Medicine Partners has entered into an agreement with Ginkgo Bioworks to be a member of their new Ginkgo Technology Network. For more details, read the press release issued by Ginkgo.

Advanced Medicine Partners CEO Andrew Knudten’s Editorial Published in Endpoints News

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An editorial written by Andrew Knudten, CEO of Advanced Medicine Partners, focused on the importance of fully characterizing a manufacturing process to promote patient safety and advance gene therapies, was published by Endpoints News. Read the full piece here.

If you want to learn more about our company’s deep experience and industry-leading expertise, reach out to us partnership@ampgtx.com.

How We Stand Apart to Deliver the Science, Partnership and Technical Solutions You Need

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At Advanced Medicine Partners, we have a very clear vision for our company – to be THE leader in process development, manufacturing, analytical development and testing for advanced therapy products. Our vision stems from our deep desire to improve the lives of patients and their families, and from our unrelenting drive to push the boundaries of science to create the purest and most potent medicines possible. To become THE leader, though, we have to clearly differentiate ourselves from traditional contract development manufacturing organizations (CDMOs). Having spent many years as clients ourselves, we identified three key areas that would set Advanced Medicine Partners apart from the rest: experience, past successes and a proven track record; depth and breadth of capabilities; and a true partnership mindset focused on accelerating programs, not slowing things down. We have been on the other side of the table, so we know how important these differences are.

We are fortunate at Advanced Medicine Partners to have one of the most experienced genetic medicines teams – at all levels, from bench to boardroom – in the industry. We boast 583 combined years of biopharma experience and 217 combined years of gene therapy CMC experience. While those numbers are impressive, it’s more important what our team accomplished in those years. For example, we have led CMC efforts for three globally approved gene therapies. Part of that work involved taking an academic manufacturing process and scaling it up for clinical and commercial needs, while working with regulators to ensure a robust CMC package and prove comparability between the academic and commercial process. We have also developed a best-in-class proprietary AAV manufacturing process that prioritizes patient safety by minimizing process and product related impurities while still maintaining industry-leading batch yields.

We’ve taken our experience and previous successes and built on them to be able to offer expansive development capabilities to partnering companies.

  • Process Sciences: We conduct both small- and large-scale development work and have completed experiments ranging in scale from small well plates to 2L and 5L benchtop bioreactors, to AMBR modular bioreactor systems, to 200L and 500L commercial-scale bioreactors. With a wide array of equipment in varying scales, manufacturers and in-process testing capabilities, we can execute early-stage bench scale development work through commercial-scale IND-enabling material generation.
  • Analytical Development: Advanced Medicine Partners has state-of-the-art analytical laboratories and equipment, which are designed to provide accurate and reliable results that meet cGMP standards. Our facilities are equipped with advanced instrumentation and technology. Our platform methods are already qualified and readily available. Many of our assays are validated and available for GMP product testing and release.
  • Quality and Regulatory Consultation: We offer a full suite of quality and regulatory consultation services. Our team has a deep understanding of successful CMC regulatory packages. We offer our partnering companies CMC strategy development, IND/IMPD CMC documentation preparation and agency support.
  • GMP Manufacturing: Advanced Medicine Partners uses a commercially viable GMP-ready process to generate product for preclinical studies, product characterization, stability, and a host of other uses including material compatibility and other tests required by regulators. We are in the process of building out a 174,000-square-foot cGMP manufacturing facility with the flexibility for clinical and commercial production across modalities. We anticipate it to be fully operational supporting clinical GMP in early 2025.

When it comes to adeno-associated virus (AAV) work, we have developed a robust and reproducible manufacturing process that is commercially viable and highly productive, including best-in-class analytics and characterization. Our innovative AAV process is one of the most productive in the industry (see figure 1).

Figure 1

There’s no doubt that experience and past successes as well as current capabilities are critical, but we think our MOST important differentiator is our partnership mindset. Traditionally, CDMO operations work independent of their clients – we want to be your partner in the truest sense of the word. We want you to think of us as your extended workbench because we know real-time data helps you drive decisions and accelerate your programs. Our partnership philosophy is fully collaborative and amplifies the capabilities of our partners. By leveraging our collective knowledge and experience (your experience and Advanced Medicine Partners’ experience), we will build a long-lasting partnership that allow for delivering fast and effective solutions for patients.

We are so proud to have launched Advanced Medicine partners to set new standard in developing and manufacturing innovative medicines. From the start, we have had our partnered companies in mind and have built our entire operation to stand apart by delivering the science, partnership and technical solutions you need. Reach out to partnership@ampgtx.com to learn more!

Advanced Medicine Partners Launches to Set New Standard for Manufacturing and Testing Advanced Medicines

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  • New company, spinning out from Jaguar Gene Therapy with funding led by Deerfield Management Company, created to provide best-in-class chemistry, manufacturing and controls (CMC) capabilities for advanced medicines, including gene and cell therapies, and address product quality and scalability issues facing many highly technical medicines
  • Experienced management and technical team who directed CMC work for numerous commercially available therapeutics, including three globally approved gene therapies
  • Operational infrastructure is in place today to support process and analytical development, quality control (QC) testing and full-scale Investigational New Drug Application (IND) enabling manufacturing – good manufacturing practice (GMP) suites are in progress for clinical and commercial manufacturing
  • Company will continue to lead CMC efforts for Jaguar Gene Therapy’s pipeline programs

DURHAM, North Carolina – January 17, 2024 – Advanced Medicine Partners, an organization that delivers process development, manufacturing, analytical development and testing for advanced therapy products, today announced its launch from Jaguar Gene Therapy with funding led by Deerfield Management Company, with additional investors including ARCH Venture Partners, Nolan Capital and others. The newly independent company is helmed by a team of experts who were directly responsible for the CMC efforts for numerous commercially available therapeutics including three globally approved gene therapies. The organization will continue to lead CMC efforts for Jaguar Gene Therapy’s pipeline programs, will support CMC work for Deerfield academic projects and private portfolio companies, and has initiated work for several biotech and pharmaceutical clients.

“Advanced Medicine Partners is a science- and mission-driven organization made up of one of the most experienced genetic medicines teams in the industry, and we are driven to deliver products and services that aim to set a new standard in the manufacturing and testing of innovative therapies,” said Andrew Knudten, CEO of Advanced Medicine Partners. “With so many biotechnology companies being slowed or halted due to manufacturing challenges including product quality and scalability, we have the experienced people and technology to deliver what’s acutely needed right now – therapeutic product with purity and potency levels that exceed expectations of partnering companies and regulators, and that will potentially bring positive benefit to patients.”

Advanced Medicine Partners has a proprietary and proven development platform and has established a robust and reproducible adeno-associated virus (AAV) manufacturing process, which is among the most productive in the industry and consistently generates industry-leading functional full capsid ratios. Advanced Medicine Partners’ process has been used with multiple genes of interest as well as multiple wild-type AAV serotypes and engineered capsids. This manufacturing process prioritizes patient safety while reducing cost of goods and will strive to set the standard for what regulatory agencies expect and patients deserve. The company has also developed a suite of platform analytical methods that can be licensed and offers assay development services.

Advanced Medicine Partners brings extensive operational experience from Jaguar Gene Therapy and other biotechnology companies, including AveXis, Novartis and Amgen. The Advanced Medicine Partners team has manufactured upwards of 350 total non-GMP batches and supplied over 20 preclinical studies, including IND-enabling efficacy and good laboratory practice (GLP) toxicology studies, as part of Jaguar. In addition to Advanced Medicine Partners’ manufacturing and analytical testing capabilities, the company offers GMP tech transfer capabilities and virtual CMC support.

Advanced Medicine Partners currently operates with more than 33,000 square feet of lab, office and warehouse space in Cary and Durham, North Carolina. The company is in the process of building out a 174,000-square-foot GMP manufacturing facility in Durham. Advanced Medicine Partners has the ability to build dedicated capacity for partnered companies in this new facility.

“We are very pleased to partner with the highly experienced team at Advanced Medicine Partners,” said Jonathan Leff, Partner at Deerfield Management Company. “With the demonstrated track record of the leadership team, the company brings much needed expertise and capability to the development and manufacturing of advanced products such as gene and cell therapies.”

ABOUT ADVANCED MEDICINE PARTNERS
Advanced Medicine Partners, LLC is driven to accelerate the development of innovative advanced medicines to enable partnering companies to make a positive difference for the patients they serve. With one of the most experienced genetic medicines teams – at all levels, from bench to boardroom – in the industry, the company uses its expertise to produce best-in-class advanced therapy products and analytics. Advanced Medicine Partners’ exclusive manufacturing platform consistently produces yields that are among the highest in the industry and purity profiles that continue to push the boundaries of what’s possible. For more information, please visit www.ampgtx.com and follow Advanced Medicine Partners on LinkedIn.

ABOUT JAGUAR GENE THERAPY
Jaguar Gene Therapy, LLC is dedicated to accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable patient populations. The company is made up of a proven team of experts who have first-hand experience in bringing novel gene therapy treatments to patients and their families. Jaguar is rapidly advancing an initial pipeline of three programs. The company’s lead program, with a pending IND, targets severe neurodevelopmental disorders caused by mutations or deletions in SHANK3 including a genetic form of autism spectrum disorder and Phelan-McDermid syndrome. The second pipeline program targets Type 1 galactosemia and the third targets Type 1 diabetes. Jaguar partners with Advanced Medicine Partners, soon to be spun out from Jaguar, to produce its therapeutics. For more information, please visit www.jaguargenetherapy.com and follow Jaguar Gene Therapy on LinkedIn.

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Contact:
Kate Neer
media@ampgtx.com
(815) 978-3891

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