Feliza Mirasol is the science editor for 

Feliza Mirasol

Following disruption in the bio/pharmaceutical supply chain as a result of the COVID pandemic, the outsourcing industry has learned to adapt and push forward in a climate that is dyamically different. In this Drug Solutions podcast episode, 

® spoke with Gil Roth, president of the Pharma & Biopharma Outsourcing Association (PBOA), and Tara Dougal, content director, Pharma portfolio, Informa Markets, on the current challenges facing the outsourcing industry today as well how contract service providers are tackling sustainability and other big trends in the industry.

 is the founder and president of the Pharma & Biopharma Outsourcing Association (PBOA), a non-profit trade group advocating for the regulatory, legislative, and general business interests of the contract manufacturing organization/contract development and manufacturing organization (CMO/CDMO) sector. He was the founding editor of 

 is content director for the Pharma portfolio at Informa Markets, where she oversees development of in-person, hybrid, and digital content for the CPHI Europe, CPHI North America, and Pharmapack Europe exhibitions and for CPHI Online.

 presents the Drug Solutions podcast, where the editors will chat with industry experts from across the pharmaceutical and biopharmaceutical supply chain. Join us as experts share insights into your biggest questions—from the technologies, to strategies, to regulations related to the development and manufacture of drug products.

Podcasts

Following disruption in the bio/pharmaceutical supply chain as a result of the COVID pandemic, the outsourcing industry has learned to adapt and push forward in a climate that is dynamically different. 

Drug Solutions Podcast: Challenges, Sustainability, and Big Trends in Outsourcing Today

Nucleic acid-based therapeutics have been stepping up into the spotlight following the deployment of messenger RNA (mRNA) COVID-19 vaccines in 2021 and the rise in clinical development programs for gene therapies. These areas of development may just be the surface of the types of therapeutic applications where nucleic acid-based therapeutics can come into play. Tackling the development challenges of these therapeutics can potentially benefit more expansive areas of unmet medical need, particularly in difficult-to-treat patient populations.

Nucleic Acid-based Therapeutics Are Stepping into the Spotlight

Pharmaceutical Technology’s The Real Message Behind Commercial mRNAProducts eBook

https://www.e-digitaleditions.com/i/1497323-pharmaceutical-technology-april-2023/0

The Real Message Behind Commercial mRNA Products


eBook: The Real Message Behind Commercial mRNA Products
April 2023
Pages: 12–17

When referring to this article, please cite it as Mirasol, F. Nucleic Acid-based Therapeutics Are Stepping into the Spotlight. 

Pharmaceutical Technology's The Real Message Behind Commercial mRNA Products 

Articles

The recent success of mRNA vaccines has opened the door for further and future development of nucleic acid-based therapies.

Avid Bioservices, a biologics contract development and manufacturing organization, has completed two expansions within the company’s mammalian cell facilities in Myford, Calif. The company has added two new current good manufacturing practice (CGMP) mammalian cell suites at the site and expects manufacturing in those suites to begin this month, according to an April 4, 2023 company press release.

The CGMP manufacturing suites include both upstream and downstream services. The additional capacity has the potential to generate approximately an additional $100 million in annual revenue, according to the press release.

In addition to launching the new suites at its Myford facility, Avid has also completed its expansion for its mammalian cell process development services, which doubles the company’s total process development capacity. Now operational, this process development expansion is expected to generate an additional $25 million in annual revenue, according to Avid in the release.

Avid Bioservices has launched new CGMP mammalian cell manufacturing suites at its Myford, Calif., facility.

Avid Bioservices Expands Mammalian Cell Capacity 

Caribou Biosciences, a US-based clinical-stage biopharmaceutical company, has received fast track designation from FDA for CB-011, an allogeneic chimeric antigen receptor T cell (CAR-T) cell therapy in development for treating relapsed or refractory multiple myeloma. The candidate is currently in Phase I development.

To Caribou’s knowledge, CB-011 is the first allogeneic CAR-T cell therapy in the clinic, the company announced in an April 4, 2023 press release. It is engineered to improve antitumor activity through an immune-cloaking strategy. Its mode of action is achieved with a beta-2-microglobulin (B2M) knockout and insertion of a B2M–HLA-E fusion protein, which blunts immune-mediated rejection.

“Fast [t]rack designation for CB-011 allows us instrumental interactions with [FDA] as we progress our clinical development and regulatory plans for CB-011. This designation could not be more timely as we recently dosed our first patient in the [Phase I] 1 trial,” said Syed Rizvi, Caribou’s chief medical officer, in the press release. “Our goal is to develop CB-011 as a readily available off-the-shelf treatment option for patients with relapsed or refractory multiple myeloma to overcome the need for apheresis or bridging therapy, variable quality and long manufacturing timelines, manufacturing failures, or the inability to bear the burden of treatments that require frequent dosing over several months.”

FDA has granted fast track designation to Caribou Biosciences for its allogeneic CAR-T cell therapy for relapsed or refractory multiple myeloma.

Caribou Biosciences Gets FDA Granted Fast Track Designation for Allogeneic CAR-T Cell Therapy

In April 2023, Ginkgo Bioworks announced two major moves that bolster its R&D and drug discovery services. The first move is the acquisition of the adeno-associated virus (AAV) capsid discovery and engineering platform assets of US-based gene therapy company, StrideBio. The second move is the formation of a partnership with the Wisconsin Alumni Research Foundation (WARF) to discover and develop next-generation chimeric antigen receptor T cell (CAR-T) cell therapies.

According to an April 5, 2023 company press release, Ginkgo will incorporate the capabilities and intellectual property (IP) it gains from the StrideBio acquisition into its end-to-end AAV gene therapy development platform. This combination will allow for the use of new tools that can effectively target many different tissue types and potentially improve the safety profile of future gene therapies. Ginkgo is also receiving StrideBio's existing library of capsids, which includes advanced candidates that have been extensively tested in large animal models and which are now available for licensing and broader partnership.

Also under the agreement, Ginkgo will gain the IP and data for StrideBio's lead preclinical asset for arrhythmogenic right ventricular cardiomyopathy , a rare genetic heart disease. Ginkgo plans to sell or outlicense this preclinical asset to a commercial partner. The transaction includes a secondary close that is scheduled upon transfer of certain additional in-license agreements to Ginkgo. Financial details of this deal were not disclosed.

"The StrideBio team has built a deep pipeline of AAV capsids and libraries to address critical challenges facing clinical gene therapy with a focus on reducing vector dose and improving safety by limiting off-target biodistribution," said Aravind Asokan, co-founder of StrideBio, in the press release.

"Combining Ginkgo's engineering and discovery capabilities in enzymes, regulatory elements, and capsids enables a holistic approach to designing an AAV gene therapy, so that we can support our partners across the entire process of designing the viral vector," said Narendra Maheshri, head of Mammalian Engineering at Ginkgo, in the press release. "We are thrilled to integrate the STRIVE platform and know-how into our foundry to augment our capabilities in capsid engineering, and we look forward to continuing to expand our existing end-to-end capabilities in AAV discovery and manufacturing."

Next-gen cell therapy discovery and development

In its collaboration with WARF, which supports research at the University of Wisconsin-Madison, Ginkgo will offer its proprietary high-throughput combinatorial CAR discovery and screening platform to researchers at WARF. The aim of the collaboration is to discover next-generation GD2 CAR-T cell therapies that have improved persistence, proliferation, fitness, and other functional properties. The goal of developing these next-gen cell therapies is to improve the efficacy of treatment for solid tumors.

According to an April 13, 2023 press release, Ginkgo will collaborate with University of Wisconsin-Madison researchers Krishanu Saha, professor at the University of Wisconsin-Madison and principal investigator (PI), and Christian Capitini, co-PI. Saha's lab is focused on developing next-generation cell engineering techniques to advance human therapeutics, while Capitini is a pediatric oncologist who has extensive clinical experience with CAR-T cell therapies. Capitini also served as a site PI for the clinical trial that led to the first FDA approval of a CAR-T cell therapy.

Under this collaboration, Ginkgo will design and characterize novel intracellular signaling domains using its pooled CAR screening platform. These signaling domains are intended to prevent the exhaustion of T cells in the GD2 solid tumor context, according to Ginkgo in the press release. Any novel designs will be validated via high throughput 

 murine models. Ginkgo and WARF also plan to collaborate on developing a pooled 

 screening platform to further advance the discovery of novel CARs.

"Ginkgo's high throughput screening method has proven successful in enabling massively parallel testing of CAR designs and has already led to the discovery of new intracellular domains (ICD) combinations," said Saha in the press release. "By partnering with Ginkgo, we aim to unlock the potential to transform patient outcomes and reimagine the future of solid tumor cancer treatment."

"This collaboration represents a fantastic opportunity to demonstrate the potential value of pooled CAR screening approaches directly in the context of devastating diseases [such as] early childhood cancers," said Narendra Maheshri, head of Mammalian Engineering at Ginkgo, in the release. "It's especially exciting to work with pioneers [such as] Professors Saha and Capitini, who have pushed R&D boundaries in the laboratory and whose approach is thoroughly grounded in the translation of their findings for clinical impact."

Ginkgo Bioworks has acquired StrideBio's AAV capsid discovery and engineering platform and has formed a partnership with WARF for development of next-gen cell therapies.

Ginkgo Bioworks Acquires AAV Capsid Technology and Forms Partnership for Next-Gen CAR-T Cell Therapies

AnaBios, a US-based preclinical contract research organization, has acquired Cell Systems, a US-based human primary cell and cell culture media company, in a deal that will bolster AnaBios’ human tissue and cells portfolio. Financial terms of the transaction were not disclosed.

The acquisition will help scientists expedite drug discovery and further understanding of cell biology through access to an expansive array of biologically relevant tools, AnaBios announced in an April 4, 2023 press release.

Cell Systems’ offering includes a variety of human primary cells, including endothelial cells from the brain, liver, kidney, eye, and lung. The company’s liver sinusoidal endothelial cells were used in a recent pivotal study of a novel liver-chip model that was said to have rigorously validated the use of the primary cells for microfluidics application in accordance with industry standards set by the International Consortium for Innovation and Quality in Pharmaceutical Development, according to the press release.

AnaBios’ advanced technologies provide human tissue samples, cells, and services for the pharmaceutical industry and academic laboratories globally. Human primary cells and tissues are important for providing clinically relevant data to increase the success rate of drug development.

“Cell Systems has decades of experience supplying our … human primary cells to pharma/biotech companies and universities around the world that enable translational research using 2D and 3D models, microfluidic devices, and in organ-chip technology. Our primary cells have been instrumental in research of the blood-brain-barrier, liver biology, kidney function, respiratory biology, and much more,” said Jesse Damm, CEO of Cell Systems, in the press release.

“AnaBios is extremely excited to provide Cell Systems’ primary cells to researchers globally and further enhance the landscape of translational research,” said Andre Ghetti, CEO of AnaBios, in the release. “By maximizing the synergies between our offerings and expertise and Cell System’s human primary cells and media, we will provide enhanced translational tools and reagents to academic, pharmaceutical, and biotech researchers and optimize their research programs through enhanced human insights.”

The acquisition of Cell Systems will bolster AnaBios’ portfolio of human tissue and cells for use in drug discovery.