We’re Headed to the 6th Annual Gene Therapy Analytical Development & CMC Summit

The Advanced Medicine Partners team will be in Boston for the 6th Annual Gene Therapy Analytical Development & CMC Summit October 30 through November 1. Stop by and see us at Booth #3!

 

6th Annual Gene Therapy Analytical Development & CMC Summit

October 30-November 1

The Westin Copley Place
10 Huntington Ave
Boston, MA 02116

Three Takeaways from My Time at Meeting on the Mesa

As I reflect on my time at Mesa last week, three main topics jump out. First, the AAV space is still in need of “competent capacity.” Sure, there is an abundance of CDMOs with plenty of large-scale bioreactors, facility space, and people available for AAV manufacturing. But as we all know, the product is the process and if your CDMO has an inconsistent process, or rapid employee turnover, or immature support systems, or just lacks experience, the process is not going to perform, and your product is going to suffer. We met with a number of potential partners that echoed this sentiment. The industry is still starving for competent capacity that can deliver the full experience for partners and patients.

This leads into my second key takeaway. As an industry, we need to continue to “raise the bar” on our expectations for both product quality and CDMO performance. Our CEO Andrew Knudten, spoke about this during his presentation last Tuesday and as I looked around the meeting room, I saw a number of heads nodding along. We cannot be complacent with the quality of products; 70% full capsids is just not good enough. The technology is there to consistently manufacture >90% functional, full capsids, so why would we ever settle for less? Our job as service providers and as drug developers is to completely remove CMC from the regulatory equation. It should be the neutral element that allows regulatory agencies to focus all their time on evaluating clinical outcomes for safety and efficacy.

Both of these items, “competent capacity” and “raising the bar,” highlight my third and final key takeaway: People + Culture is still a winning formula. The equipment you have, the facility you build, the systems you implement, they’re all dependent upon the people who utilize them. You can’t have “competent capacity” if you don’t have the right people executing. You can’t “raise the bar” on product quality and performance if you don’t have the right leaders demanding it. Even then, if you’ve been able to find the right people, you can’t sustain excellence unless you foster a culture that diffuses throughout the company. Culture is the foundation that will support the company through the lifecycle of ups and downs and sustain dedicated employees. It’s the one true asset that cannot be duplicated. Throughout Mesa, we had people stopping by our booth complementing our team, informing us that they heard about us, that they wanted to work with a team like us. It’s probably overused but understated: People + Culture = Hard to beat.

In closing, I’d like to comment on my experience as a whole while out in Phoenix this week. ARM does an outstanding job hosting this event. Everything from the venue to the catering, the stage presentation, the speakers, the networking events; it is all top notch. I attend a number of these large industry conferences, and this one is at the top of the list. So, thank you to the ARM team and to everyone else who came out to make Mesa another successful meeting. Here’s to seeing everyone again next year – and sweating it out together in the desert heat!

Will Jones
Senior Vice President, Analytical Development and QC

Taking an Industry Pulse at Meeting on the Mesa

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

  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.

There’s No Substitute for Deep Analytical Experience

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 CEO Andrew Knudten’s Editorial Published in Endpoints News

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

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!