iECURE Enters Agreement with Center for Breakthrough Medicines (CBM) to Supply Materials for Future Clinical Programs
Partnership to focus on manufacturing clinical materials for future clinical studies
PHILADELPHIA–(BUSINESS WIRE)–iECURE, a gene editing company focused on mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need, and Center for Breakthrough Medicines (CBM) today announced the companies have entered into a strategic collaboration wherein CBM will produce and supply Good Manufacturing Practices (GMP)-grade adeno-associated virus (AAV) for use in iECURE’s future clinical studies to enable development of iECURE’s programs with the shared mission of accelerating the availability of these potential treatments to the patients.
“Our approach to gene editing relies on highly complex manufacturing processes, and we are eager to establish partnerships to ensure a reliable supply of GMP materials for future clinical development,” said Paul Firuta, Chief Operating Officer of iECURE. “CBM was able to offer us significant amount of guaranteed capacity with scheduling flexibility and we are confident that they will be a strong partner with their state-of-the-art facilities and end-to end capabilities.”
“iECURE’s approach to gene editing is quite innovative and has the potential to bring significant hope to patients and families facing devastating diagnoses,” said Audrey Greenberg, Chief Business Officer and Co-Founder of CBM. “Our ability to provide customizable clinical and commercial GMP manufacturing solutions integrated with industry-leading comprehensive in-process testing, quality control, and lot release programs allows us to support our partners like iECURE through the entirety of their product lifecycle and is aligned with our mission to bring life changing medicines to patients in need.”
iECURE is a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of monogenic liver disorders with significant unmet need. We believe our approach has the potential to replace and restore the function of a dysfunctional gene by knocking-in a healthy copy, regardless of mutation, to offer durable gene expression and long-term, potentially curative, therapeutic benefit. Our management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products. We intend to leverage our team’s core strength in research and development strategy to identify what we believe to be the most suitable target and modality for our product candidates to address particular liver diseases. We are collaborating with the University of Pennsylvania’s Gene Therapy Program, or GTP, led by James M. Wilson, M.D., Ph.D., to utilize GTP’s world-class translational expertise and infrastructure, which has helped generate our initial pipeline of potential product candidates. For more information, visit www.iecure.com and follow on LinkedIn.
About Center for Breakthrough Medicines (CBM)
CBM is a cell and gene therapy contract development and manufacturing organization (CDMO) based in the heart of Philadelphia’s Cellicon Valley. CBM offers pre-clinical through commercial manufacturing capabilities including process development, plasmid DNA, viral vector, cell therapy and a full suite of testing and analytical capabilities. Through a single-source, end-to-end solution, CBM accelerates time to market without compromising quality.
Center for Breakthrough Medicines Beats Back on the 2022 Biotech Bear Market with Rapid Hiring and Expansion
“Since its launch, The Center for Breakthrough Medicines (CBM) has seen exponential growth as a concierge life sciences services organization. That growth was highlighted during the first quarter of this year with the hiring of approximately 200 additional people.
Audrey Greenberg, co-founder of CBM and also the executive director of The Discovery Labs, said the successful growth seen at CBM is a testament to the great company culture the organization has built since its founding in 2019. Among the hires in the first quarter of this year is John Lee, PhD, the new Vice President and Head of Cell Therapy. Lee, an accomplished CAR-T immunobiologist, was hired in March to lead cell therapy programs at CBM. Prior to joining CBM, Lee established the Cell Therapy Platform team at Janssen Pharmaceuticals. He also served as head of Oncology Cell Therapy at GlaxoSmithKline.
Lee reports to CBM’s Chief Executive Officer Joerg Ahlgrimm, who was brought on to build out the organization’s capacity to support the manufacturing of cell and gene therapies. Ahlgrimm, who previously served as chief operations officer for the organization, joined CBM after serving as Head of Global Operations Pharma Biotech and Nutrition at Lonza.”
Considerations To Get Your CAR T Testing Strategy Right
“Less than five years after the first approval of a CAR T therapy in 2017, momentum for CAR T and other cell therapies has never been greater.
Given their experience reviewing and approving six CAR Ts across 10+ indications, the FDA released a draft guidance “Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products” in March of 2022. As a trusted partner to cell therapy product developers such as Achilles Therapeutics, we at the Center for Breakthrough Medicines (CBM) identified three considerations – testing CAR-T starting material, establishing manufacturing process controls, and implementing robust testing analytics – that our clients prepare for as part of their analytical development prior to finalization of the guidance. This will help to monitor and report on the right critical quality attributes (CQAs) of their CAR T products which is needed for characterization of the product’s safety and efficacy that is reported to health authorities.”
Philadelphia region’s 71% increase in VC funding easily tops all major U.S. markets during 1Q
“Philadelphia-area startups saw the greatest percent increase in venture capital funding in the first quarter, according to analytics firm CB Insights, an increase that more than doubled any other major metropolitan region….
Greater Philadelphia was one of three regions where venture capital funding didn’t drop from the fourth quarter of 2021. The first-quarter figure was bolstered by a $350 million investment from SK Inc. in King of Prussia’s Center for Breakthrough Medicines.”
Process Platforms vs. Custom Process Development for AAVs and Gene Therapies
“Gene therapies hold huge promise for patients with a range of monogenic diseases and unmet medical needs. However, they’ve had their fair share of safety concerns – dosing and delivery have been the sources of recent setbacks. Efficient, scalable, and cost-effective manufacturing is another hurdle the industry needs to overcome before commercially viable therapies can be licensed. AAV technology advances are also needed to address yield, material costs, and cycle times in a bid to bring down COGS and ultimately increase financial accessibility by global patient populations.
There is also a cash crunch echoing through the gene therapy industry with layoffs and company closures affecting many promising late-stage advanced therapies from commercializing. This appears to be the sharp end of a wider industry trend; the hype bubble has burst, and the promise of steady, high-valuation cash influxes has gone.
To address issues of cost, time to market and manufacturing complexity, many developers have turned to turnkey manufacturing platforms. The question is whether these platforms provide the right solution for successful CMC development. Use of platforms has come full circle – 5-10 years ago many developers were manufacturing their viral vectors at academic vector core centers. This led to academic processes that were not scalable or suitable for commercial GMP manufacturing, resulting in a big shift for biotech to develop processes that they own and control.”
Advanced Therapy Manufacturing
Cell and gene therapies represent an emerging paradigm, and the future of advanced therapies. As of early 2022, the U.S. Food and Drug Administration (FDA) has approved four gene therapies1 and six cell-based treatments.2 Two additional gene therapies are approved in the EU.
Approvals only tell part of the story, however. More than 1600 gene therapies3 are currently in clinical trials, and the FDA has to date received more than 900 applications to initiate gene-based treatments.4 The agency expects that by 2025 it will issue upwards of 200 investigational new drug (IND) applications for advanced therapies per year, with approval of up to 20 such treatments. This unprecedented growth comes with unique challenges for advanced therapy developers and manufacturers, which often center around the three essentials of quality, speed and innovation.