Susan Haigney

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On Oct. 10, 2023, the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) announced the launch of the Viral Vectors NIIMBL-led program. The program will be focused on developing available, economically viable manufacturing processes and analytical platforms for adeno-associated virus (AAV) gene therapy vectors. NIIMBL hopes the program will provide access to high-quality viral vectors without cost or speed limitations.

The program will include a process workstream with shared access to an AAV process platform for rare diseases and advanced process performance. It will also include an analytical workstream to enhance access and utility of an AAV analytical toolkit and reference resources. A parallel initiative in workforce development for AAV vector manufacturing and analytics will also enhance the program by educating and training personnel. “Advancing the industry is not just about creating new technology and making it available; you need the people and knowledge to apply that technology most effectively,” said Eric Hacherl, Viral Vector Program co-lead, in a press release. “Our hope is that as the technology is developed, we will have sufficient training resources in place and be able to establish a capable and confident workforce, ensuring there is no delay in technology adoption.”

“There are thousands of rare diseases, many of which could be candidates for AAV-based gene therapy. Collectively advancing and converging around core manufacturing and analytical technologies so we can avoid redundant work and consistently achieve better outcomes is fundamental to bringing more therapies to more patients faster,” said Guangping Gao, PhD, member of the NIIMBL-led Viral Vector Program Steering Committee and gene therapy pioneer from the University of Massachusetts Chan Medical School, in the press release.

“We have assembled an initial steering team of thought leaders and subject matter experts over the past year and have identified two key workstreams to kick-off this program. Time is our biggest motivator, and we know that moving quickly is essential to ensuring these advances remain relevant to the treatment needs of patients,” said Tim Charlebois, Viral Vector Program co-lead, in the release.

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The program will improve access to adeno-associated virus gene therapy vectors.

NIIMBL Launches Viral Vector Program

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Sharp, a packaging and clinical trial supply services company, announced on Oct. 5, 2023 the acquisition of Berkshire Sterile Manufacturing (BSM), a contract development and manufacturing organization (CDMO) located in Massachusetts that offers clinical and commercial sterile injectable products. Sharp anticipates the acquisition will allow the company to offer fully integrated small-to-medium scale sterile injectable services.

Sharp currently offers clinical supply, packaging, and distribution solutions. BSM’s sterile manufacturing expertise and CDMO services will complement Sharp’s services, according to the company, offering customers services from sterile drug manufacturing to clinical trial services, commercial packaging, and distribution. BSM manufactures small-scale sterile clinical and commercial drug products and has fill/finish services that include isolator-based sterile filling of vials, syringes, and cartridges with lyophilization capabilities.

“The market continues to demand expertise and capacity in sterile manufacturing due in part to the growth in rare and orphan disease therapies. With sterile injectables a huge part of addressing this unmet medical need, the acquisition of BSM allows Sharp to provide a fully integrated solution for our biopharma clients that is a natural extension of our existing injectables and clinical packaging expertise,” said Kevin Orfan, president and CEO of Sharp, in a press release. “This acquisition, which builds on the existing partnership between our two companies, will greatly benefit our clients, creating an efficient manufacturing and distribution service for biotech companies developing sterile injectable products across the world.”

Dr. Shawn Kinney, BSM’s co-founder and CEO, who will join Sharp’s Senior Leadership Team, stated in the press release, “This is an exciting development for everyone at BSM and for our clients, who will benefit from a seamless downstream packaging, labeling, storage, and distribution service through Sharp’s network of facilities and expertise. With Sharp’s support, I am looking forward to the opportunity to grow the sterile manufacturing business and to broaden our reach in the service of our clients and their patients.”

With the acquisition, Sharp plans on offering fully integrated small-to-medium scale sterile injectable services.

Sharp Acquires Berkshire Sterile Manufacturing

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Clene, a late clinical-stage biopharmaceutical company focused on treatments for neurodegenerative diseases, announced on Oct. 5, 2023 that it has been awarded a $45.1 million four-year grant by the National Institute of Neurological Disorders and Stroke (NINDS). The grant will support an Expanded Access Protocol (EAP) for Clene’s amyotrophic lateral sclerosis (ALS) drug, CNM-Au8. An EAP is an FDA-regulated pathway that allows people with a serious and life-threatening disease to access an investigational drug that is not yet approved by FDA. In addition, Clene will continue its ongoing ALS EAP programs.The grant is part of theAccelerating Access to Critical Therapies for ALS Act (ACT for ALS) that was signed into law on Dec. 23, 2021. The Act called for increased support for development and access to new ALS treatments.

Led by Jinsy A. Andrews, MD, MSc, FAAN, of Columbia University; Eric Anderson, MD, PhD,MBA, FAAN, of Synapticure; and Benjamin Greenberg, MD, MHS, FAAN, Head of Medical of Clene, the EAP will allow ALS patients to participate through Synapticure’s telemedicine neurology clinic and a network of nationwide clinics. “This study will monitor safety, survival, and clinical worsening as well as levels of key biomarkers related to disease progression with CNM-Au8 treatment,” according to the company.

“This EAP study will give ALS patients who don’t meet the criteria to enroll in a clinical trial an opportunity to try CNM-Au8 as a novel investigational therapy through this EAP program,” said Jinsy A. Andrews, M.D., MSc, FAAN, an associate professor of neurology in the Division of Neuromuscular Medicine and director of Neuromuscular Clinical Trials at Columbia University Vagelos College of Physicians and Surgeons, in a press release. “Programs like this help to advance research and much-needed innovation in ALS.”

"We are truly excited to be a part of this grant with Clene and Columbia and to support people living with ALS by providing access to treatments that could meaningfully impact the course of their disease. We are grateful to NINDS for recognizing how a virtual platform like Synapticure can provide expanded access programs in a remote capacity. It will allow us to reach people living with ALS across the U.S. who have not previously had access to investigational medicines, like CNM-Au8," said Dr. Anderson, VP of Clinical Operations and Care Delivery at Synapticure, in the release.

“Clene has demonstrated evidence of consistent safety and improved survival for CNM-Au8 across a broad ALS population in two independent Phase II trials and an ongoing EAP with up to 3.8 years of follow-up. This new EAP provides access to CNM-Au8 for more people living with ALS and enables the collection of survival, safety and biomarker data in a population not studied in clinical trials. These data can help provide confirmatory support for the existing trial data Clene has gathered in its clinical trials,” said Dr. Greenberg, in the release.

Clene was awarded the four-year grant for the National Institute of Neurological Disorders and Stroke to support an Expanded Access Protocol for ALS drug.

NIH Awards $45.1 Million Grant for ALS Treatment

Dyadic International, a biotechnology company that builds microbial platforms, announced on Sept. 26, 2023 a collaboration with bYoRNA SAS, a French biotechnology company developing scalable mRNA bioproduction platforms. The development and commercialization agreement the companies have entered into includes work on disruptive bioproduction technologies to use the therapeutic potential of messenger RNA (mRNA).

“I am excited to announce this development and commercialization agreement to explore the potential to utilize our C1 protein production platform to produce high-quality messenger RNA products at lower cost, which will be easier to manufacture than 

 mRNA, making it accessible to everyone, including patients from poorer countries. The global mRNA vaccine and therapeutics market was valued at US$34.7 billion in 2022 and is expected to reach US$68.1 billion by 2030, making this an extremely valuable potential opportunity for both companies to enter the rapidly expanding market for mRNA-based vaccines and therapies,” said Mark Emalfarb, Dyadic’s chief executive officer, in a press release. “If this collaboration is successful, Dyadic has the potential to receive certain defined milestone and royalty payments from bYoRNA and the ability to obtain a license to the BRNA [bio RNA] platform,” Mr. Emalfarb concluded.

“The COVID-19 pandemic brought mRNA technology to the forefront, showing its potential to prevent and treat diseases. Within this collaboration, we will combine our respective technologies to try to express ‘bio’ RNA from Dyadic’s eukaryotic C1 cells, to design, develop and commercialize messenger RNA products,” said Pascal Viguié, bYoRNA’s chief executive officer, in the release.

“We believe that by combining bYoRNA’s novel eukaryotic ‘bio’ RNA platform with Dyadic’s industrially proven C1 protein production platform, we will be able to provide the pharmaceutical industry with a more cost-efficient platform for manufacturing large quantities of lower cost mRNA, enabling access to mRNA vaccines and drugs to a broader global population,” said Thierry Ziegler, bYoRNA’s chief technology officer, in the press release.

The companies plan on targeting the production of abundant, low-cost messenger RNA from C1-cells.

Dyadic and bYoRNA Collaborate on Production of mRNA

FDA announced on Sept. 22, 2023 that VistaPharma is voluntarily recalling one lot of Sucralfate Oral Suspension, 1g/10mL, because of contamination of the product by 

. The product, which is used as an antiulcer therapeutic and distributed nationwide, is packaged in a 16 oz (414 mL) polyethylene terephthalate bottle with NDC 66689-305-16. The recalled lot is number 810300 with an expiration date of Oct. 31, 2023.

Microbial contamination of the oral suspension may result in life-threatening infections, such as endocarditis and necrotizing soft tissue infections, according to a press release. As of the time of the press release, the company had not received any reports of adverse events. Adverse events may be reported to FDA via the agency’s MedWatch Adverse Event Reporting program.

The recall is being undertaken by a contractor, Inman, which is notifying distributors and arranging for the return of recalled product.

VistaPharm is recalling Sucralfate Oral Suspension, 1g/10mL because of Bacillus cereus contamination.

Sucralfate Oral Suspension Recalled Due to Microbial Contamination

The European Medicines Agency (EMA) gave an update on Sept. 15, 2023 about its work with the European Network for Health Technology Assessment (EUnetHTA 21). The consortium worked with the agency to deliver a number of milestones in preparation for the Regulation on Health Technology Assessment, which was entered into force in January 2022 and will apply as of January 2025. The regulation will “govern the European cooperation between medicine regulators and HTA bodies. Under the new framework, EMA and HTA bodies will collaborate in the context of joint clinical assessments, joint scientific consultations, and the identification of emerging health technologies,” according to EMA.

The agency noted several achievements that happened as part of its cooperation with EUnetHTA 21, including seven parallel joint scientific consultations for medicines, addressing challenges for advanced therapy medicinal products in oncology, organizing training for those involved in HTA processes, and recommending optimization of assessment reports. The EUnetHTA 21 stopped operating on Sept. 16, 2023, but implementation of the regulation will continue under the HTA Coordination Group, according to EMA. A new framework for Parallel EMA/HTA Scientific Advice from September 2023 until January 2025 has been established by EMA and HTA organizations.

“EMA’s collaboration with EUnetHTA began in 2010 as a project to test out whether early engagement between regulators and HTA bodies could bring tangible benefits for patient access to medicines,” said Michael Berntgen, head of Scientific Evidence Generation Department at EMA, in a press release. “Together, we were able to help medicine developers enhance clinical research and become more efficient in generating the evidence relevant for both regulatory authorities and HTA bodies.”

“Although the EMA-EUnetHTA cooperation is drawing to a close, the road does not end here,” said Niklas Hedberg, chair of the EUnetHTA 21 Consortium executive board, in the press release. “We are very proud to see that our cooperation has now been enshrined into European law. The experience gained provided essential technical input to shape the new legislation. In fact, we continue our joint work and will do this on a new footing, with more participants, additional responsibilities, and high expectations from stakeholders.”

The agency’s work with EUnetHTA 21 helped it to prepare the EU for implementation of the regulation.