Univercells and Andelyn Biosciences Form Separate Drug Development Partnerships

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Editor's note: this story was originally published in PharmTech.com.

On Mar. 25, 2024 Univercells announced a collaboration with Altamira Therapeutics, a producer of nanoparticle-based technology, under which Univercells will use Altamira’s SemaPhore nanoparticle platform to deliver messenger RNA (mRNA) vaccines. Also on Mar. 25, Andelyn Biosciences, a contract development and manufacturing organization (CDMO), announced a partnership with Grace Science, a biotechnology company focused on developing novel therapies based on N-Glycanase 1 (NGLY1), an enzyme critical to proteostasis.

Nanoparticle-delivered mRNA vaccines

Using Altamira’s SemaPhore nanoparticle platform, Univercells will first test an in-vitro and in-vivo proprietary mRNA vaccine. If successful, the companies plan to negotiate a commercial agreement for the development and manufacture of nanoparticle-based mRNA vaccines using Univercells’ production platform.

“We’re delighted to be partnering with Altamira to explore better ways to deliver mRNA to patients,” said José Castillo, PhD, Chief Technology Officer of Univercells, in a Univercells press release. “mRNA vaccines, and mRNA in general, have proven to be a game-changer in how we prevent, treat, and cure diseases in a range of fields from oncology to infectious diseases. To unlock its full potential, however, we need constant innovation to make mRNA products more effective, efficient, and affordable. One key step is to develop platforms that use lower doses of mRNA.”

Novel therapies for NGLY1 deficiency

Andelyn Bioscience and Grace Science are partnering to tech transfer and manufacture GS-100, which is a suspension process adeno-associated viruses (AAV) NGLY1 gene therapy. GS-100 is being developed to treat patients with NGLY1 deficiency. The aim of this partnership, according to Andelyn, is to maximize program efficiency, support the ongoing clinical trial, and accelerate the therapy to patients.

NGLY1 deficiency is a life-threatening disease that has no approved therapy, according to a company press release. NGLY1 is a cytosolic enzyme critical to removing N-linked glycans of misfolded N-glycoproteins. The enzyme is part of endoplasmic reticulum-associated degradation (ERAD), a component of protein quality control in which misfolded and potentially toxic proteins in the endoplasmic reticulum are eliminated. Patients with this deficiency suffer from debilitating symptoms such as cognitive impairment, developmental delay, hypo alacrimia, movement disorders, and other neurological issues.

GS-100, a recombinant AAV9 vector that encodes a full-length version of the human NGLY1 gene, was granted orphan drug designation by FDA and the European Medicines Agency in 2021. FDA also granted the therapy rare pediatric disease designation in 2021 and fast-track designation in 2023. Andelyn’s knowledge of AAV processes and late-stage manufacturing are a key part of the upcoming partnership, which is focused on quality control while also accelerating Grace’s manufacturing timelines.

Matt Wilsey, CEO and co-founder of Grace Science stated in an Andelyn press release, “We are very excited about the partnership between Grace Science and Andelyn Biosciences. Andelyn’s deep expertise and experience in AAV gene therapy manufacturing will assure that our NGLY1 patients have access to safe, high-quality drug product for our clinical trial and beyond.”

“Both Andelyn and Grace Science value strong collaboration and share a customer-centric focus and quality-first mindset,” added Matt Niloff, chief commercial officer at Andelyn Biosciences, in the press release. “These synergies provide patients and their families confidence and hope for success of the GS-100 program.”

Sources: Univercells, Andelyn Biosciences, National Library of Medicine