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Drug Solutions Podcast: 

Pharmaceutical packaging must advance to adapt to new, complex modalities

Out-of-the-Box Packaging Advancements

From Theory to Reality: Automating Process Development

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The Importance of Appropriate Supplier Oversight

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Feliza Mirasol is the science editor for 

Frontier Scientific Solutions, a temperature-controlled storage and transportation company that exclusively serves the life sciences industry, announced on Nov. 13, 2024 that it has established state-of-the-art current good manufacturing practice (CGMP) facilities at Wilmington International Airport (ILM) in North Carolina, United States, and Shannon Airport (SNN) in County Clare, Ireland. The strategic locations will enable the company to create the first life sciences-dedicated direct global gateway, according to a company press release. Supported by a $1.5 billion commitment from an investor, the gateway is central to Frontier Scientific Solutions’ goal to create streamlined shipping lanes—starting with a route between North America and Europe—specifically for time- and temperature-sensitive pharmaceutical and biopharmaceutical products.

These facilities are supported by the $1.5 billion funding from GID, a real estate investor, owner, operator, and developer. Frontier Scientific Solutions expects that the facilities in both locations will bring further financial and supply chain management benefits through designation as Free Trade Zones (FTZ). The company unveiled plans at ILM for the scale and impact of its global gateway model. The company expects that this model will enable companies in the life sciences industry to transport time- and temperature-sensitive materials in significantly shorter timeframes. Benefits include fewer touchpoints, less risk, and more sustainable operations.

"We are thrilled to announce this transformative partnership with the airports at Wilmington and Shannon, enabled by GID's investment,” said Steve Uebele, CEO of Frontier Scientific Solutions, in the company press release. “Our initiative disrupts the life science supply chain market—promoting zero product losses, increasing sustainability, and directly saving lives—and it marks the first step in Frontier's goal to become the market-leading solutions provider."

The funding will be used for the construction of CGMP-compliant, temperature-controlled storage facilities housed at Shannon and within the FTZ at Wilmington. Having FTZ designation will facilitate streamlined customs and FDA processing, duty deferral, and operational efficiencies, all of which are critical for the efficient and secure distribution of life sciences products across international borders, the company stated in the release.

"GID’s commitment underscores our dedication to developing cutting-edge infrastructure that meets the specialized needs of the life sciences industry,” said Greg Bates, president and CEO of GID, in the release. “The new facilities in Wilmington and Shannon will create an unparalleled logistics network between the US and Europe, empowering companies to deliver critical healthcare products with unmatched speed and precision."

Based on live case studies conducted by Frontier Scientific Solutions, it was consistently demonstrated that a reduction of days to weeks in the transportation and waiting times for temperature-sensitive shipments were achieved with its gateway model. “Providing the industry with direct distribution makes this global gateway model faster, reducing exposure to temperature excursions, risk from unnecessary touchpoints, and carbon emissions. It will set a new benchmark for quality, speed, and environmental responsibility,” the company stated in the press release.

The facilities at ILM and SNN will be linked by a direct lane dedicated exclusively to life sciences materials. Flight operations are slated to begin early in 2025. Frontier Scientific Solutions also offers a fast direct-ocean lane from North Carolina to Cork, Ireland, and can achieve delivery in 12 days via this route. This new infrastructure will allow the company to disrupt the market by enabling faster deliveries while reducing reduced risk.

In addition to supply chain logistics, the biopharma industry is seeking ways to minimize risks in providing temperature-sensitive molecules to patients, such as 

innovating product formulation and drug delivery systems

Prioritizing Formulation Strategies for Temperature-Sensitive Biotherapeutics

Pharmaceutical Technology Trends in Formulation

Articles

Frontier Scientific Solutions has unveiled plans to use a $1.5 billion commitment from a major investor to create a global free trade zone gateway to facilitate the life sciences supply chain using Wilmington International Airport in North Carolina.

Frontier Scientific Solutions Plans a Global Free Trade Zone Gateway to Supply Life Sciences Industry on Back of $1.5 Billion Investment

Felicity Thomas is Associate Editorial Director of Pharmaceutical Technology, Pharmaceutical Technology Europe, BioPharm International.

Eisai Europe and Biogen have received a positive opinion from the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) for the use of their amyloid-beta (Ab) monoclonal antibody (mAb) lecanemab in treating adult patients with a clinical diagnosis of mild cognitive impairment and mild dementia due to Alzheimer’s disease (early AD). The positive opinion, announced by Eisai in a Nov. 15. 2024 press release, was primarily based on data from a global Phase III clinical trial (1).

Patients in the European Union (EU), who will be eligible for treatment once the European Commission has made its final decision on the treatment, would be those who are apolipoprotein E ε4 (ApoE ε4) non-carriers or heterozygotes with confirmed amyloid pathology. ApoE is a protein that is involved in the metabolism of fats in humans and has been identified as the strongest genetic risk factor for sporadic AD and an important biomarker to determine disease susceptibility (2).

“[The] positive CHMP opinion brings us one step closer to being able to offer a potential treatment option which targets an underlying cause of AD to eligible patients in the EU for the first time,” said Gary Hendler, regional chairman and CEO, Eisai EMEA, senior vice-president and global corporate officer, Eisai Co. Ltd, Tokyo, in the press release (1). “AD is a progressive neurodegenerative disease that poses significant challenges to human health and wider society—the loss of someone’s memory and independence can have a significant impact on not only those living with the disease, but also their family and friends. Eisai is committed to making a meaningful difference to all those affected by AD, and we are pleased that our ongoing work with the CHMP is helping to make this available to eligible patients in the EU.”

CHMP’s opinion has been sent to the European Commission for its final review, with a decision on the marketing authorization application expected within 67 days of receipt of the opinion. Lecanemab is already approved in the United States, China, Japan, South Korea, Hong Kong, Israel, the United Arab Emirates, and Great Britain, and is under review in 17 countries.

“The positive CHMP opinion marks a significant step and recognizes the potential of this medicine to make a difference to eligible individuals and their families impacted by this disease. We look forward to the EC’s decision and are pleased to be one step closer to offering this medicine in the EU,” added Wolfram Schmidt, president, head of Europe, Biogen, in the press release (1). “As a company, we are dedicated to furthering Alzheimer’s disease research and treatment, aiming to help address the unmet needs in this devastating condition.”

Lecanemab (brand name Leqembi) is a humanized immunoglobulin gamma 1 (IgG1) mAb that selectively binds to soluble Ab aggregates (protofibrils) and insoluble Ab fibril aggregates. According to research, the reduction of Ab protofibrils has been highlighted as potentially useful in reducing the progression of AD (3).

1. Eisai. Eisai Receives Positive Opinion from the CHMP in the European Union for Lecanemab in Early Alzheimer’s Disease. Press Release, Nov. 15, 2024.
2. Pires, M.; Rego, A.C. ApoE4 and Alzheimer’s Disease Pathogenesis—Mitochondrial Deregulation and Targeted Therapeutic Strategies. 

 (1), 778.
3. Ono, K.; Tsuji, M. Protofibrils of Amyloid-b are Important Targets of a Disease-Modifying Approach for Alzheimer’s Disease. 

CHMP has granted Eisai Europe and Biogen a positive opinion for the use of their monoclonal antibody therapy in treating early Alzheimer’s disease.

EMA’s CHMP Gives Positive Opinion for Use of Lecanemab in Early Alzheimer’s Disease

Avantor, a provider of mission-critical products and services to the life sciences and advanced technology industries, announced on Nov. 14, 2024 that it has opened its new flagship Bridgewater Innovation Center in Bridgewater, NJ.  Spanning 60,000 square feet, the state-of-the-art R&D facility joins the company’s network of 13 research and innovation centers globally.

The expanded facility doubles the company’s previous laboratory and pilot plant capacity and was purpose-designed to enable collaborative work. The center houses spaces for upstream and downstream process development, dedicated analytical testing labs, and a viral vector laboratory. It also houses an expanded pilot plant that supports scale-up simulations, which is intended to enable rapid customization across the entire bioprocessing workflow, according to a company press release.

“Our new Bridgewater Innovation Center is twice the size of our previous facility and adds capacity, talent, and capabilities to support the growing demands for monoclonal antibodies [mAbs], cell and gene therapy [CGT], and mRNA [messenger RNA] workflows,” said Michael Stubblefield, president and CEO of Avantor, in the press release. “Working side-by-side with customers in our network of innovation centers across the globe enables us to power science at every step to deliver life-changing therapies faster and more cost effectively.”

The center gives Avantor the capabilities to optimize and accelerate biomanufacturing processes at scale. Advantages include faster problem solving, streamlined knowledge exchange, and strategic co-innovation. The facility is staffed with PhD scientists, bioengineers, biologists, and process engineers, which enhances capacity and expertise to support customers' needs. As stated in the press release, “The facility also supports Avantor’s sustainability goals, reinforcing the [c]ompany’s commitment to environmental responsibility.”

The center is aimed at helping biopharmaceutical companies accelerate the development of vital therapies across multiple modalities, including mAbs, CGTs, and protein/peptide therapeutics. Expansive collaboration spaces built throughout the facility allow scientists, biologists, and bioengineers to work side-by-side with biopharma manufacturers. These collaborations leverage the company’s advanced products, real-time analytical data, and process knowledge to optimize processes and materials. Having this co-development environment saves crucial time and resources, according to the company in the press release.

® (1), Benoit Gourdier, executive vice-president and head, Bioscience Production Segment, Avantor, noted how the pace of innovation in biopharma has been posing challenges, particularly in relation to which platform technology to use. “This is something where we’re seeing a fast evolution. From that perspective, at Avantor we are agnostic because we can provide product, solutions, technologies, platforms, support, and this innovation center is a good example. No matter the modality, we have a solution for the players,” Gourdier said in the interview.

Nandu Deorkar, senior vice-president, Bioscience Production Research & Development, Avantor, also spoke in the interview and pointed out that, because of the shift to personalized medicine manufacturing, there will be more distributed manufacturing. “If you look at the cell and gene therapy [space], [patients] will be treated on a personal basis, so there will be more distributed manufacturing on a local basis; so how do we support this geographically?” Deorkar said in the interview (1).

Meanwhile, the anticipated flood of new biotherapeutic modalities onto the market in the near future aimed at precision treatment is expected to open up many new opportunities for service providers, Gourdier said in the interview (1).

Avantor Sees Future of Opportunities with Growing Development of Next-Gen Biotherapeutics

Avantor's newly launched expanded Innovation Center in Bridgewater, NJ, is designed for integrated workflows and features purpose-built collaboration spaces. 

Newly Opened Innovation Center in New Jersey Gives Avantor Advantage in Solving Life Sciences Challenges

In preparation for the launch of its new innovation center in Bridgewater, NJ, Avantor executives discussed a future full of opportunities for service providers, which will come from a growing number of next-generation biotherapeutics in the development pipeline that are anticipated to reach the market in the near future.

“The first thing [that comes to mind] is lots of opportunities, because this is really going back to the base of innovation,” said Benoit Gourdier, executive vice-president and head, Bioscience Production Segment, Avantor, in an interview with 

® at a press event held at the Bridgewater facility on Nov. 13. 2024.

Where once the biopharma industry was dominated by monoclonal antibodies (mAbs), the industry can now expect to see a wave of newer, more innovative therapies aimed at achieving precision treatment. “Starting 25-30 years ago, it was really mAbs, mAbs, mAbs, and traditional vaccines,” Gourdier said, adding, “We grew up in this environment. Now we have this diverse portfolio of modalities, so [that will provide] lots of opportunities to go after, to learn.”

The challenges that Gourdier anticipates in the future could likely revolve around chemistry, fluid handling, meeting high purity in a regulated market, among others, but Gourdier is confident that Avantor will be well prepared to meet these challenges and to offer the appropriate support as a service provider.

Nandu Deorkar, senior vice-president, Bioscience Production Research & Development, Avantor, added that, because of the shift to personalized medicine manufacturing, there will be more distributed manufacturing. “If you look at the cell and gene therapy [space], [patients] will be treated on a personal basis, so there will be more distributed manufacturing on a local basis; so how do we support this geographically?” Deorkar said in the interview.

Deorkar also added, “Some of these therapies have 48 hours to 72 hours injection requirement after manufacturing, so [not all] the manufacturing can be done [in one place].”

Gourdier, meanwhile, pointed out that, in addition to the expectation of a different manufacturing and supply chain scenario for next-gen biotherapeutics, the industry suffered from supply chain disruptions due to the COVID-19 pandemic, which are still ongoing in the post-COVID environment. Regionalization has become more important, he noted. “[Developers] want global partners with regional focus,” he stated.

Other factors that have disrupted the pace of development for these next-gen biotherapeutics has been a drop in funding as a direct result of the COVID-19 pandemic, Gourdier added. “Most of the big players are okay,” he observed, “but for smaller players, the amount of money available for them has decreased significantly. We are just [coming] back [from that]. Now we are in modest recovery from that (i.e., the funding) perspective.”

Meanwhile, the pace of innovation has itself been posing challenges, particularly in relation to which platform technology to use. “This is something where we’re seeing a fast evolution. From that perspective, at Avantor we are agnostic because we can provide product, solutions, technologies, platforms, support, and this innovation center is a good example. No matter the modality, we have a solution for the players,” Gourdier stated.

Avantor’s new Bridgewater Innovation Center is set to launch on Nov. 14. It has been designed as a state-of-the-art research and development facility and joins the company’s network of 13 research and innovation centers globally.

Executives at Avantor talk about the future of the biopharmaceutical industry and the anticipated impact that a wave of next-generation biotherapeutics will bring.

Chris Spivey is director of Industry Relations and Strategic Partnership, MJH Life Sciences.

Brian Feth and Jonathan Grinstein go behind the headlines to discuss the impact of a Republican administration on Federal Trade Commission actions, tax relief, and Health and Human Services (HHS) leadership; as well as advancements in CAR-T.

Behind the Headlines: What the US Election Results Mean for Industry, In Vivo CAR-T Applications, and more

 is a bi-weekly panel discussion examining the latesttrends, readouts, and other factors that drive pharmaceutical news and innovation.Each episode features consultants, venture capitalists, scientists, patient advocates, and journalists discussing the prior weeks’ top news while seekingto highlight the more enduring lessons that hide behind the headlines.

In episode 6, Brian Feth, CEO atXcell Biosciences, and Jonathan Grinstein, North American Editor, 

, go behind the headlines to understand how a recently elected Republican administration in the U.S. might spur investment into the biopharmaceutical industry, particularly through tax breaks and loosening of certain regulations. They also discuss how stem cell research might be slowed and how nontraditional leadership of HHS might influence vaccine policy and pandemic preparedness.

FDA approval of Autolus Therapeutics' obecabtagene autoleucel

 (Aucatzyl) for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) (1). This autologous CAR T-cell therapy, targeting CD19-positive B-cells, has shown promising results in the FELIX trial, with a notable complete remission rate. However, in line with FDA’s current warnings for treatment-related secondary cancer, “The approval includes a boxed warning for risks of cytokine release syndrome (CRS), immune effector cell–associated neurotoxicity syndrome (ICANS), and potential T-cell malignancies.”(1) The panel also noted Autolus’ other updates, including the first dosing of patients in their phase 1 CARLYSLE trial of obecabtagene autoleucel in refractory systemic lupus erythematosus (SLE)(2).

Sticking to CAR-T breakthroughs for B-cell abnormalities, the panel also pointed out that Interius BioTherapeutics dosed the first-ever patient with an in vivo CAR gene therapy for the treatment of B-cell malignancies. This is a possible game-changer for advanced therapies as in vivo therapies remove many of the lengthy and expensive manufacturing steps currently required for cell and gene therapies. Interius president and CEO, Phil Johnson, MD, said, “This milestone marks the first time that a durable in vivo CAR therapy has been used in the clinic. INT2104 has the potential to overcome ex vivo CAR therapy challenges with a single-dose, off-the-shelf, widely accessible therapy for the treatment of B cell malignancies enabled by Interius’ programmable platform.” (3)

1. FDA approves obecabtagene autoleucel for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. FDA. November 8, 2024. Accessed November 8, 2024. 

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-obecabtagene-autoleucel-adults-relapsed-or-refractory-b-cell-precursor-acute

2. Autolus Therapeutics reports third quarter 2024 financial results and business updates. Press release. Autolus Therapeutics. November 12, 2024. 

https://autolus.gcs-web.com/news-releases/news-release-details/autolus-therapeutics-reports-third-quarter-2024-financial

3. Interius BioTherapeutics doses first patient with in vivo chimeric antigen receptor (CAR) gene therapy for B-cell malignancies. Press release. Interius BioTherapeutics. October 23, 2024. 

https://interiusbio.com/2024/10/interius-biotherapeutics-doses-first-patient-with-in-vivo-chimeric-antigen-receptor-car-gene-therapy-for-b-cell-malignancies/

Videos

Behind the Headlines: What the U.S. Election Results Mean for Industry, In Vivo CAR-T Applications, and more 

Ginkgo Bioworks, a provider of a cell programming platform and biosecurity, announced on Nov. 12, 2024 that it has completed the first milestone of a previously announced partnership (1) with Merck, known as MSD outside of the United States and Canada. Their collaboration aims to improve biologics manufacturing, and, according to the terms of their agreement formed in August 2023, Ginkgo will receive a research milestone payment of $9 million in cash for completing this milestone.

Under their partnership, Ginkgo uses its expertise and capabilities in cell engineering, ultra-high-throughput multiplexed screening, protein characterization, and process optimization to improve biologics production efficiency and increase yields. The company is eligible to receive up to $490 million in aggregate of upfront research fees, research milestone fees, option license payments, and commercial milestone payments (1).

"We're excited to achieve the first milestone of our collaboration with Merck and continue our work to improve biologic manufacturing production," said Jason Kelly, CEO and co-founder, Ginkgo Bioworks, in a company press release. "It's very meaningful when our team can drive impactful and technically innovative work like this, especially when we're working shoulder-to-shoulder with counterparts like the team at Merck.”

The two companies will now move on to the second stage of the partnership, which involves focusing on the continued improvement of the biologics production process.

“As we go into the next phase of this project, we'll continue to deploy the strain engineering, high-throughput screening, and process development capabilities as well as the assets and know-how that make Ginkgo a unique and powerful partner to top pharmaceutical companies," Kelly said in the release.

The August 2023 partnership deal is the second collaboration between Ginkgo and Merck. In October 2022, the companies entered into a biocatalysis project to improve Merck's API manufacturing (2). Under the biocatalysis deal, Ginkgo earned an upfront research and development fee and was eligible for success-based research and development milestone payments. Ginkgo was also eligible to earn commercial milestone payments for each of a specified number of biocatalysis targets. These targets have the potential to total, in the aggregate, up to $144 million, according to a company press release issued at that time.

"At Merck we are always seeking new and innovative ways to optimize process efficiency while maintaining product integrity," said Michael Kress, PhD, senior vice-president, development sciences and clinical supply, Merck Research Laboratories in an Aug. 7, 2023 press release (1). "We are pleased to build upon our relationship with the Ginkgo Bioworks team."

1. Ginkgo Bioworks. Ginkgo Bioworks Announces Collaboration with Merck to Improve Biologic Manufacturing. Press Release. Aug. 7, 2023.
2. Ginkgo Bioworks. Ginkgo Bioworks Announces Collaboration with Merck to Improve Active Pharmaceutical Ingredient Manufacturing. Press Release. Oct. 11, 2022.

Under an ongoing partnership with Merck, known as MSD outside of the United States and Canada, Ginkgo Bioworks has achieved the first milestone in a project aimed at improving biologics production.

Merck and Ginkgo Bioworks’ Collaboration to Improve Biologics Manufacturing Has Reached Its First Milestone

TrakCel, a provider of integrated cell and gene therapy (CGT) orchestration solutions, announced on Nov. 12, 2024 that some manufacturers have selected its orchestration platform, OCELLOS, to support the management of five out of seven autologous and matched allogeneic cell therapy products. These products are approved or are expected to be approved in 2024. TrakCel’s technologies enable the precise management, control, and tracking of CGTs.

An IT portal, such as OCELLOS, is needed to successfully navigate the CGT supply chain, as manufacturers require tools to help launch these FDA-approved products and ensure that approved treatment centers (ATCs) can administer the CGT safely and effectively, according to a company press release.

“We’re committed to driving forward patient access to ground-breaking CGTs and are delighted to be supporting a growing number of manufacturers as they navigate the CGT supply chain and successfully launch their product,” said Fiona Withey, PhD, CEO, TrakCel, in the press release. “Commercializing CGT products is a complex and challenging process that requires close collaboration between manufacturers and ATCs. However, as more products are approved, ATCs are concerned this could lead to further proliferation of custom-made orchestration platforms, creating an additional burden on their staff that could potentially hinder the success of future new product launches.”

A list of CGTs approaching regulatory approval by the end of 2024 are listed in a presentation given by The Alliance for Regenerative Medicine (ARM) in August 2024 (1). OCELLOS is a Salesforce-powered solution that was specifically designed for the CGT industry, according to TrackCel, which stated in its press release that the platform aims to deliver a unique therapy process by connecting base modules and configurable forms with real-time visibility showing the progress of each personalized therapy through the supply chain. This workflow is intended to support efficiency in the supply chain and ease collaboration with ATCs.

“Working with our Industry Advancement Board (IAB), we recognize that there is a growing need to standardize on one platform to help foster a consistent approach to capabilities and communication processes, training, workflows, and use of terminology,” Withey stated in the press release. “OCELLOS is a proven technology ready to drive industry-wide standardization and ensure life-enhancing therapies reach the patients that so desperately need them.”

TrackCel also pointed out in its release that it sees continued investment in custom-built IT portals, even as CGT manufacturers are increasingly using OCELLOS. The company stated that these IT portals help facilitate product ordering and patient enrollment as well as efficient orchestration of supply chain activities, including manufacturing and logistics.

“As more CGT products are approved … an increasing number of custom-built platforms could overwhelm clinical site staff and be extremely expensive for each manufacturer to build and support, potentially limiting access to the therapy, and impacting patient safety,” TrackCel warned in its press release.

1. ARM. Sector Snapshot: The Emerging Value of Cell and Gene Therapy. 

alliancerm.org/sector-snapshot-august-2024

Manufacturers of five autologous or matched allogenic cell therapy products have selected TrakCel's  IT platform, OCELLOS, to orchestrate the administration of these therapies, which are approved or expected to be approved in 2024.

TrakCel’s OCELLOS Platform Selected to Support Treatment Management of Five Upcoming Cell Therapy Products

Ascend Advanced Therapies announced on Nov. 12, 2024 that it is partnering with EW Healthcare Partners to expand capabilities and invest in infrastructure in the United States. As part of the agreement, EW Healthcare’s Advanced BioScience Laboratories (ABL), a biotherapy, oncolytic and viral vector contract development, and manufacturing organization (CDMO) located in Rockville, Md., will become part of Ascend. EW Healthcare will also make capital investment in Ascend.

ABL’s Rockville facility and team will provide development and manufacturing experience, virus modalities, and good manufacturing practice (GMP) capacity for adeno-associated virus (AAV) production. Ascend’s fill/finish capacity in the US will also be expanded.

“We founded Ascend with a vision to quilt together the best operators and technologies within the advanced manufacturing field. The complementary services at ABL and Ascend will further strengthen our service offering to customers,” Fred Cohen, chairman of Ascend and Founding investor at Monograph Capital, said in the press release (1). “We look forward to working with our new colleagues at the Rockville facility and our partners at EW.”

“We have known Ascend and followed its progress in creating effective and high-quality gene therapies, and have been impressed with how the management team, which we also backed at Cognate BioServices, has expanded the business,” said Evis Hursever, PhD, managing director of EW Healthcare Partners. “We are excited about merging ABL with Ascend to create a scalable CDMO that will serve as a global platform for the manufacturing of advanced therapies. We have considerable experience and expertise in this space and the combined company’s strategic plans are very well aligned with our long-term vision. We look forward to working with the Ascend and ABL teams to further growth.”

This isn’t the first investment Ascend has made in 2024. In April, the company acquired Beacon Therapeutics GMP facility in Alachua, Fl. to obtain Beacon’s chemistry, manufacturing, and controls team; process and analytical development capabilities; and three ocular gene therapy products utilizing AAV to deliver gene constructs that address retinal disorders.

“Ascend was founded with a unique science and technology-driven strategy. As we continue building the most flexible [AAV] platform across serotypes on the market, we also understand the critical value of our experienced employees,” Mike Stella, CEO at Ascend, said in a press release (2). “This acquisition allows us to now support customers from research through to commercialization and adds to one of the most experienced gene therapy teams in the market. Every day we are working together to grow the company with a long-term mindset of quality first to appropriately balance quality and yield for the life of each product.”

“We are excited to enter this strategic arrangement with Ascend to advance our sight saving therapies toward approval. This alignment is critical to securing GMP drug product supply for our late-stage clinical asset AGTC-501 for the treatment of X-[l]inked [r]etinitis [p]igmentosa,” David Fellows, CEO at Beacon Therapeutics, said in the release. “The Ascend team brings significant experience in gene therapy manufacturing and a strong commitment to building capabilities to support future Beacon pipeline programs.”

1. Ascend. Ascend Advanced Therapies Partners with EW Healthcare Partners to Expand United States Capacity and Capabilities. Press Release. Nov. 12, 2024.
2. Ascend. Ascend Acquires GMP Manufacturing Capacity in Alachua, Florida from Beacon Therapeutics. Press Release. April 9, 2024.

Ascend is partnering with EW Healthcare Partners to expand capabilities in the United States.

Ascend Advanced Therapies Expands US Footprint in New Partnership

AAPS PharmSci360 Challenges in Peptide-Based Drugs

The development of oral peptide-based drugs presents unique challenges, specifies Brittany Hayes, PhD, Highly Potent & Oncology Platform Director at CordenPharma, due to the harsh environment of the gastrointestinal tract, highly acidic stomach, and enzymes that degrade peptides. “New manufacturing technologies and formulation techniques have shown to increase bioavailability by a few percentage points,” says Hayes. “But there's a lot of variability here, including variability from one patient to the next and also in the same patient from one day to the next day. One of the big ones that's been out there is permeation enhancers.”

Analytics is an important factor for the development of peptide-based drugs, highlights Hayes, because degradation products may be difficult to detect and quantify. “And then, with the use of these permeation enhancers, there needs to be simultaneous dissolution of the permeation enhancer and the peptide,” she says. “So, there may be some challenges here with analytical methods.” She also points out these novel excipients may require more regulatory qualifications and could be treated differently than conventional excipients.

Click the video above to watch the full interview.

Pharmaceutical Technology® caught up with Brittany Hayes, PhD, Highly Potent & Oncology Platform Director at CordenPharma, after AAPS PharmSci 360 to talk about the challenges and quality requirements involved in the development and manufacture of peptide-based oral dosage forms. 

AAPS PharmSci 360: Challenges in Peptide-Based Drugs

Text sign showing Industry News. Business photo text delivering news to the general public or a target public | Image Credit: © Artur - © Artur - stock.adobe.com

The University of Texas MD Anderson Cancer Center announced on Nov. 8, 2024 that it is creating the Institute for Cell Therapy Discovery & Innovation for the development of cell therapies for cancer, autoimmune diseases, and infections. The institute will bring scientists and clinicians together to provide insight on immunology and cell engineering with $80 million support from philanthropic organizations and institutions.

Led by Katy Rezvani, vice president and head of the Institute for Cell Therapy Discovery & Innovation, the new institute will integrate MD Anderson’s different research areas with the goal of accelerating discovery and development of new cell therapies from preclinical through clinical phases. This effort includes the James P. Allison Institute and Institute for Data Science in Oncology. External researchers and bio/pharmaceutical companies will also be part of the collaborative effort.

“Cell therapies have delivered profound results for patients with hematologic cancers, but there remain too many patients that do not benefit. It is critical to our mission to bring these promising therapies forward as options for patients with cancer and other conditions,” Rezvani said in a press release (1). “Through our discoveries, we aim to provide many more patients with effective cell therapies that are available as off-the-shelf treatment options, limiting the need for lengthy hospital stays and associated costs.”

“MD Anderson clinicians and researchers have been instrumental in shaping the cell therapy field, and we are proud to take this important step into the future,” said Peter WT Pisters, president of MD Anderson, in the release. “The Institute for Cell Therapy Discovery & Innovation is a key piece of our institutional strategy, as we focus on cancer breakthroughs to improve patient outcomes. Our world-class facilities, exceptional talent and unique research environment will enable our teams to deliver transformational new medicines and to further our efforts to maximize our impact on humanity to finally end cancer.”

Cell therapies use a patient’s immune cells as building blocks to create treatments. MD Anderson has performed clinical trials on FDA-approved chimeric antigen receptor (CAR) T cell therapy products targeting CD19 and BCMA for B-cell lymphomas, leukemias, and multiple myeloma. The institute also advanced CAR natural killer (NK) cells into clinical studies patients with lymphoid malignancies, based on cord blood-derived CD19 CAR NK cell therapies developed by Rezvani and her team. Additional research by the group has been done for cell therapies in solid tumors, including adoptive T-cell receptor therapy targeting MAGE-A4 for patients with multiple solid tumor types.

“For years, our talented scientists and clinicians have been at the forefront of discovery, understanding and development of novel cell therapies,” said Giulio Draetta, chief scientific officer at MD Anderson, in the release. “By integrating with experts across the research and clinical enterprise, the Institute for Cell Therapy Discovery & Innovation will be able to create important new treatments, tailored to unmet patient needs, in ways only possible here.”

MD Anderson. MD Anderson Launches Institute for Cell Therapy Discovery & Innovation to Deliver Transformational New Therapies. Press Release Nov. 8, 2024.

The new Institute for Cell Therapy Discovery and Innovation will utilize expertise to develop cell therapies for cancer, autoimmune diseases, and infections.

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