Batavia Biosciences Announces Collaboration with Phoenix Nest Inc. to Accelerate Development of Therapeutics for Sanfilippo Syndrome

Batavia Biosciences Announces Collaboration with Phoenix Nest Inc. to Accelerate Development of Therapeutics for Sanfilippo Syndrome

Woburn, MA, 16 October 2023 — Batavia Biosciences, a leading global Contract Development and Manufacturing Organization, announced a new partnership with Phoenix Nest Inc., a bespoke biotech company focusing on treating ultra-rare diseases. The collaboration aims to fast-track the development and production of recombinant human N-acetylglucosamine-6-sulfatase (rhGNS) for treatment of Sanfilippo syndrome, a rare genetic disorder.

Batavia Biosciences will perform crucial process and analytical development for Phoenix Nest Inc. This work will enable the future production of rhGNS for toxicity studies and establishing a scalable, Good Manufacturing Practice (GMP)-compatible process.

“We are very pleased that Phoenix Nest decided to contract Batavia Biosciences based on our proven track record and technological expertise. We are honored to collaborate and provide solutions for patients battling Sanfilippo syndrome.” says Peter Abbink, PhD, Managing Director at Batavia Biosciences, Inc., located in Woburn, MA.

The need for high-quality, specialized therapeutics for Sanfilippo syndrome remains an urgent priority. Affected individuals often face severe neurological symptoms with limited treatment options. This collaboration aims to advance the scientific understanding and accelerate the development of treatments for this devastating disease.

“Phoenix Nest is developing therapies for Mucopolysaccharidosis type III (MPS III), also referred to as Sanfilippo Syndrome. We made significant progress towards the development of an enzyme replacement therapy for Sanfilippo syndrome type D, including an observations study [NCT05648851] in MPS IIID patients. We are excited about our collaboration with Batavia for our enzyme manufacturing and scale-up development” stated Jill Wood, CEO, Phoenix Nest Inc.

About Phoenix Nest Inc.

Phoenix Nest Inc. is a bespoke biotechnology company specializing in developing innovative therapies for ultra-rare diseases, with a primary focus on Sanfilippo syndrome. Sanfilippo syndrome (Mucopolysaccharidosis III), a set of genetic neurodevelopmental disorders that result in progressive neurological, developmental, and physical regression. The impacted individuals require life-long medical and supportive care as there are no approved cures or treatments.  Phoenix Nest is the proud recipient of several Small Business Innovation Research grants from the National Institute of Health. Through funding from the NIH under Award Number U44NS089061, we have been able to facilitate the research in academic labs and licensed these programs for development and translation to clinical interventions.

About Batavia Biosciences

Batavia Biosciences significantly contributes to ease human suffering from infectious diseases, cancer, and rare diseases by leveraging our innovative technologies and in-depth know-how. We do this for viral vaccines and cell and gene therapies at all stages of the development trajectory, from the transition into early clinical studies, to navigating late-stage clinical development and entering commercial manufacturing at higher speed, reduced costs, and with increased success. The company operates state-of-the-art development and manufacturing facilities in Europe and the US and, as part of the CJ CheilJedang global family, is building a strong position in global health with its strategic partners worldwide.

Media Contacts

Phoenix Nest Inc.

Jill Wood, CEO

Batavia Biosciences

Peter Abbink, Managing Director

Batavia partners in consortium to develop vaccine against Rift Valley fever 

Batavia partners in consortium to develop vaccine against Rift Valley fever 

Batavia Biosciences is proud to be a key partner in an important initiative aimed at combating the deadly Rift Valley fever. We are thrilled to share that as part of the consortium working alongside esteemed partners such as Wageningen Bioveterinary Research, Bunyavax BV, CR2O BV, University of Veterinary Medicine Hannover, and Integrum Scientific, LLC, we are stepping up our efforts to bring a protective vaccine to East Africa, where Rift Valley fever is endemic.

Batavia’s role as consortium partner

Our role in this endeavor is pivotal. At Batavia, we strive to reduce human suffering from infectious disease by helping improve vaccine affordability and availability around the world. Therefore, we have specialized in developing highly intensified manufacturing processes, leveraging our cutting-edge HIP-Vax® platform to create vaccines with a low cost of goods. As a consortium partner, our primary focus is on developing a scalable manufacturing process that can be rapidly scaled-up in the event of an outbreak. Furthermore, we are honored to take on the responsibility of manufacturing the clinical material for the Phase I and IIa trials planned for East Africa.

This initiative is a significant milestone in our ongoing battle against infectious diseases, particularly Rift Valley fever. The project, generously supported with up to US$25.9 million in funding from CEPI and backed by the European Union’s Horizon Europe program, will accelerate the development of a much-needed protective vaccine in East Africa.

Rift Valley fever: A climate-driven disease with epidemic potential

Rift Valley fever is a potentially lethal virus that can be transmitted to humans through mosquito bites or contact with infected livestock. With climate change increasing the risk of RVF outbreaks, the need for a protective human vaccine has never been more urgent. As a ‘One Health’ priority, the development of safe and effective human vaccines for RVF is crucial, and we are committed to playing our part in this vital endeavor.

Together with our esteemed partners and the support of CEPI and the European Union, we are determined to make a difference in the fight against Rift Valley fever.

Read the full press release for more details: CEPI expands partnership with Wageningen Bioveterinary Research to advance clinical trials of Rift Valley fever vaccine in affected countries – CEPI

Batavia Biosciences Joins the Alliance for Regenerative Medicine 

Batavia Biosciences Joins the Alliance for Regenerative Medicine 

Batavia Biosciences is excited to announce its new Alliance for Regenerative Medicine (ARM) membership. ARM is a premier international advocacy organization that brings regenerative medicines and advanced therapies to patients worldwide. By joining ARM, Batavia Biosciences is committed to advancing the cell and gene therapy field. 

“By aligning ourselves with the Alliance for Regenerative Medicine, we are strengthening our commitment to the broader cell and gene therapy community. Our primary objective is to help innovators develop and deliver ground-breaking therapies that can change and save lives,” says Peter Abbink, PhD, Managing Director at Batavia Biosciences, Inc., located in Woburn, MA. “Our extensive expertise in bioprocessing makes us a valuable partner in accelerating the journey of these therapies from the lab to the market. We aspire to be a key collaborator in a consortium where we can substantially contribute to enhancing the quality of life for patients and caregivers.” 

Batavia Biosciences has a strong track record of offering state-of-the-art development and manufacturing solutions for viral vectors and is an integral player in the cell and gene therapy field. Through its cutting-edge HIP-Vax® platform technologies and services, Batavia Biosciences facilitates the rapid development and scalable manufacturing of life-changing therapies, while significantly reducing the cost of goods. Innovative bioprocess intensification technologies pave the way for significantly improved production of viral vector products, used in gene therapy and immuno-oncology. 

About The Alliance for Regenerative Medicine 

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization championing the benefits of engineered cell therapies and genetic medicines for patients, healthcare systems, and society. As a community, ARM builds the future of medicine by convening the sector, facilitating influential exchanges on policies and practices, and advancing the narrative with data and analysis. We actively engage key stakeholders to enable the development of advanced therapies and modernize healthcare systems so patients benefit from durable, potentially curative treatments. 

As the global voice of the sector, we represent more than 400 members across 25 countries, including emerging and established biotechnology companies, academic and medical research institutions, and patient organizations. 

Batavia: Lentivirus for first patient NL

It is with great enthusiasm and excitement that we would like to congratulate Leiden University Medical Center and the research team on their recent accomplishment using the lentivirus. Researchers have successfully treated a baby with stem cell gene therapy for severe combined immunodeficiency disease (SCID). This form of SCID (which often claims the life of the victim before finishing their first year of life), had previously never been treated with gene therapy and now the disease has been successfully cured.

Batavia Biosciences is proud to announce that we were able to play a role in helping reduce human suffering as Batavia produced the lentivirus vector for this incredible project. Professor Frank Staal references the lentivirus vector as the “wheelbarrow that brings the gene to the right place” within the article. It’s apparent that amazing work is being done by the top names in the gene therapy field, and we are honored to be able to contribute with our expertise in viral vectors, specifically, the lentivirus in this project.

Wilfried Bakker, Director of Science and Innovation, states, “To be part of this development, we could not be prouder. Our contribution of the lentivirus vector to treat this form of SCID means a lot to us, but even more to the patient and future patients with this life-threatening form of SCID.”

Read the full press release with the links provided:

Volkskrant Article 
LUMC Press Release

Batavia Biosciences Optimizes Global Quality Assurance with Veeva Vault Quality Suite

Batavia Biosciences Optimizes Global Quality Assurance with Veeva Vault Quality Suite

BARCELONA, Spain — September 15, 2022- Veeva Systems  (NYSE: VEEV) today announced Batavia Biosciences selected  Veeva Vault Quality Suite  to optimize its global quality assurance (QA), unifying content, processes, and training management on a single platform. Batavia will also benefit from real-time insights that support ongoing QA improvements and inspection readiness.

“As our development footprint grew globally, we needed to optimize our QA approach to better meet future needs,” said Jolanda Van Vliet, director of quality assurance, Batavia Biosciences. “Bringing our QA onto Veeva’s cloud-based platform enables us to centrally manage quality documentation and training, facilitate collaboration among authors, and improve reaction time to quality events.”

Based in the Netherlands,  Batavia Biosciences  is a full-service contract development and manufacturing organization (CDMO) specializing in viral vaccines, virotherapy, and protein vaccines. With  Veeva Vault QMS, Batavia can centrally manage QA processes to better ensure GxP compliance, while  Veeva Vault QualityDocs  automates workflows and audit trails, simplifying document management, updates, and review processes.  Veeva Vault Training  will further help the CDMO ensure role-based qualifications.

“It is a privilege to partner with Batavia to support their launch of a fully digital quality assurance system and boost inspection readiness,” said Sofia Lange, director strategy, Vault Quality & Manufacturing at Veeva. “With the addition of Vault Quality Suite, Batavia now has a scalable foundation that will strengthen its QA processes and oversight across operations.”

Additional Information

For more on Veeva Vault Quality Suite, visit:
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About Veeva Systems

Veeva is the global leader in cloud software for the life sciences industry. Committed to innovation, product excellence, and customer success, Veeva serves more than 1,000 customers, ranging from the world’s largest pharmaceutical companies to emerging biotechs. As a Public Benefit Corporation, Veeva is committed to balancing the interests of all stakeholders, including customers, employees, shareholders, and the industries it serves. For more information, visit

Gene therapy developers see an expanded viral toolbox

There are still many challenges in gene therapy analytics and manufacturing according to gene therapy developers. Our Global Technical Director,  Alfred Luitjens  was interviewed on this topic by Genetic Engineering & Biotechnology News. The interview was in response to his presentation on the manufacturing of a lentiviral vector for a gene therapy against RAG1-SCID at the PepTalk conference 2020.

Link to  full  article.

Low-cost viral vector manufacturing

High-throughput screening for viral vectors

Viral vector manufacturing

Maximizing protein expression