Pharmaceutical Technology Editors

CordenPharma announced on May 13, 2021 that it plans to expand its non-GMP capacity at its Centre of Excellence for Peptide Process Development in Frankfurt, Germany, and increase its upstream capacity at its CordenPharma Colorado site with the introduction of a new 3000-L solid phase peptide synthesis (SPPS) vessel.

The capacity expansions fall in line with CordenPharma’s strategy to provide its customers with quick and economically appealing solutions, the company said in a press release. In addition to the new SPPS vessel, CordenPharma plans to add a second 100-cm high-pressure chromatography column at its CordenPharma Colorado site.

“This additional column will provide the capacity to purify several tons of peptide annually, which in conjunction with our existing 100 and 80-cm columns, gives us more flexibility and redundancy to maximize supply security for all of our partners,” said Matthieu Giraud, PhD, global director of the Peptides, Lipids & Carbohydrates Platform, CordenPharma, in the press release.

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The expansions will add peptide manufacturing capacity.

CordenPharma Announces New Capacity Expansions at its US and Germany Sites

Biogen and Capsigen, a US-based adeno-associated virus (AAV) vector development company, announced on May 10, 2021 that they have entered into a strategic research collaboration to engineer novel AAV capsids to deliver gene therapies that address the underlying genetic causes of various central nervous system (CNS) and neuromuscular disorders for up to $1.37 billion.

Under the terms of the agreement, Capsigen will receive a $15 million upfront payment and is eligible to receive up to $42 million in potential research milestones, with an additional $1.25 billion in potential development and commercial payments, Biogen said in a company press release. Biogen will obtain an exclusive license under Capsigen’s proprietary technology for an undisclosed number of CNS and neuromuscular disease targets through the collaboration.

Additionally, as part of the agreement, Capsigen will utilize its TRADE platform and associated technologies to develop and classify novel AAV capsids designed to meet disease-specific transduction profiles.

“Through this collaboration, we aim to solve key technological challenges in the delivery of gene therapies to target tissues. One of our priorities for technology innovation is the discovery of AAV capsids with improved delivery profiles,” said Alfred Sandrock, Jr., MD, PhD, head of Research and Development at Biogen, in the press release. “We are investing for the long-term by building platform capabilities and advanced manufacturing technologies with the goal of accelerating our efforts in gene therapy.”

“At Capsigen, we believe the next revolution in gene therapy will be driven by engineered AAV capsids designed to meet disease-specific transduction profiles,” added John Bial, CEO, Capsigen, in the press release. “Biogen is a leader in neuroscience, and we are excited for the opportunity to work with them to potentially bring new treatments to patients. This collaboration is consistent with our strategy to work with world-class companies to develop the next generation of gene therapies.”

Biogen and Capsigen have entered into a strategic research collaboration to engineer novel AAV capsids to deliver gene therapies that address the underlying genetic causes of various central nervous system and neuromuscular disorders. 

Biogen and Capsigen to Develop Novel AAV Capsids for up to $1.37 Billion

On May 11, 2021, Fujifilm Irvine Scientific celebrated 50 years of providing cell culture media products and services.

Originally established in the 1970s as a fetal bovine serum supplier, the company continuously proved its strength in the cell culture solutions space by modernizing embryo culture with commercially available media for 

 fertilization clinics worldwide and by bringing chemically defined media to biotherapeutic manufacturers, Fujifilm Irvine Scientific said in a company press release. Recently, the company introduced products better suited for cell culture-based vaccine production, and advanced cell and gene therapy applications.

“We have reflected on our trailblazing history and many contributions over the last five decades. Our 50th year coincided with unprecedented challenges from the COVID-19 pandemic,” said Yutaka Yamaguchi, CEO and chairman of the board, Fujifilm Irvine Scientific, in the press release. “We are grateful for the dedication and commitment shown by our employees to uphold our standards of delivering the highest quality products and service under these difficult conditions. We are also humbled by the extraordinary efforts of the community we serve in the fight against the virus and honored that our customers trust Fujifilm Irvine Scientific to provide the critical cell culture media needed to manufacture lifesaving therapies and vaccines, and to support medical applications. We are inspired to continue our mission to improve health through innovation, partnership, and service for the next 50 years.”

Originally established in the 1970s as a fetal bovine serum supplier, Fujifilm Irvine Scientific continuously proved its strength in the cell culture solutions space.

Fujifilm Irvine Scientific Celebrates 50 Years of Providing Cell Culture Media Products and Services

The National Institutes of Health (NIH) reported on May 11, 2021 that research has found that an investigational gene therapy developed by researchers from the University of California, Los Angeles and Great Ormond Street Hospital in London can restore the immune systems of infants and children who have severe combined immunodeficiency due to adenosine deaminase deficiency (ADA–SCID), a rare, life-threatening inherited immunodeficiency disorder.

Caused by mutations in the ADA gene that impair the activity of the adenosine deaminase enzyme needed for healthy immune system function, ADA–SCID is often treated with enzyme replacement therapy, which does not reconstruct immune function, must be taken once or twice weekly, and requires a genetically matched donor, NIH said in a company press release. The new research evaluated the gene therapy by implanting a normal copy of the ADA gene into the patient’s own blood-forming stem cells. Then, a vector delivered the normal ADA gene to these cells in the laboratory and the genetically corrected stem cells are infused back into the patient, who has received a low dose of the chemotherapy medication busulfan to let the cells establish themselves in the bone marrow and begin producing new immune cells.

Phase I/II clinical trials conducted in the United States and the United Kingdom showed the gene therapy to be safe overall, with some participants experiencing mild or moderate side effects attributed to the chemotherapy they received.

“These findings suggest that this experimental gene therapy could serve as a potential treatment option for infants and older children with ADA–SCID,” said Anthony S. Fauci, MD, director of NIH’s National Institute of Allergy and Infectious Diseases, in the press release. “Importantly, gene therapy is a one-time procedure that offers patients the hope of developing a completely functional immune system and the chance to live a full, healthy life.”

NIH reported that an investigational gene therapy developed by researchers from the University of California, Los Angeles and Great Ormond Street Hospital in London can restore the immune systems of infants and children who have severe combined immunodeficiency due to adenosine deaminase deficiency. 