Pharmaceutical Technology Editors

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.”

Articles

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. 

Research Shows Gene Therapy Restores the Immune Systems of Infants and Children with Rare Immunodeficiency

FDA expanded the emergency use authorization (EUA) for the Pfizer-BioNTech COVID-19 vaccine to include adolescents 12–15 years of age, according to a May 10, 2021 announcement. The vaccine will be administered in two doses, three weeks apart, using the same dosage and dosing regimen for people ages 16 and older.

In an ongoing clinical trial of 2260 participants, with half receiving the vaccine, the immune response measured in 190 participants, 12­–15 years of age, was at least as good as the immune response of 170 participants, 16 through 25 years of age.

An analysis of cases of COVID-19, conducted seven days after the second dose, showed no cases of COVID-19 occurred among 1005 vaccine recipients, demonstrating the vaccine was 100% effective in preventing COVID-19.

Side effects in adolescents were consistent with those reported in clinical trial participants 16 years of age and older.

FDA amended the EUA originally issued on Dec. 11, 2020 for administration in individuals 16 years of age and older. From March 1, 2020 through April 30, 2021, the Centers for Disease Control and Prevention (CDC) received reports of approximately 1.5 million COVID-19 cases in individuals 11–17 years old.

“The FDA’s expansion of the emergency use authorization for the Pfizer-BioNTech COVID-19 vaccine to include adolescents 12 through 15 years of age is a significant step in the fight against the COVID-19 pandemic,” said Acting FDA Commissioner Janet Woodcock. “Today’s action allows for a younger population to be protected from COVID-19, bringing us closer to returning to a sense of normalcy and to ending the pandemic. Parents and guardians can rest assured that the agency undertook a rigorous and thorough review of all available data, as we have with all of our COVID-19 vaccine emergency use authorizations.”

Adolescents are now eligible for the Pfizer-BioNTech COVID-29 vaccine under expanded EUA.

FDA OKs Pfizer-BioNTech Vaccine for Adolescents

Eli Lilly and Company (Lilly) and MiNA Therapeutics, a UK-based RNA activation therapeutics company, announced on May 11, 2021 that they are launching a global research collaboration to develop novel drug candidates using MiNA's proprietary small activating RNA (saRNA) technology platform.

Under the terms of the partnership, MiNA will receive a $25-million upfront payment and will be eligible to receive development and commercialization milestones of up to $245 million per target as well as tiered royalties from the low-single to low-double digits on product sales resulting from the collaboration, Lilly said in a company press release. Through the collaboration, MiNA will use its saRNA platform to research up to five targets selected by Lilly to address diseases across its key therapeutic focus areas, while Lilly will handle the preclinical and clinical development of the candidates and will maintain exclusive commercialization rights for any products resulting from the collaboration.

"Small activating RNAs are a promising new technology, which will expand the breadth of Lilly's RNA therapeutics platform and the targets we can pursue," said Andrew C. Adams, PhD, vice-president for new therapeutic modalities at Lilly, in the press release. "We are excited about the potential of combining MiNA's leading saRNA platform and our expertise in new modalities to accelerate development of RNA-based medicines in areas of high unmet medical need."

"This collaboration with Lilly is an important validation of our saRNA platform," added Robert Habib, CEO of MiNA Therapeutics, in the press release. "Lilly's expertise in the field of RNA therapeutics and clinical development will greatly enhance our efforts to realize the technology's full potential. Together, we aim to unlock new targets in multiple therapeutic areas and to ultimately move them towards clinical development and commercialization."

Through the collaboration, MiNA will use its saRNA platform to research up to five targets selected by Lilly to address diseases across Lilly's key therapeutic focus areas.

Lilly and MiNA Therapeutics to Collaborate on Novel Drug Development

Sterling Pharma Solutions and OncoTEX, an oncology company and member of the iQ Group Global bioscience investment enterprise, The iQ Group Global, announced on May 10, 2021 that they have entered into a clinical development agreement for the manufacturing of OncoTex’s novel platinum-resistant oncology drug candidate, OxaliTEX, for use in clinical trials.

Through the agreement, Sterling will produce clinical trial material at its current good manufacturing practice (CGMP) facility in Germantown, Wis., which can accommodate highly potent active pharmaceutical ingredients in fully contained manufacturing suites, Sterling said in a company press release. OxaliTEX has been developed using OncoTEX’s oncology drug platform, TEX Core, to address the clinical need for ovarian cancer diagnosis and treatment.

“The current prognosis for patients with platinum-resistant ovarian cancer is very poor, so the development of OxaliTEX is an important step in advancing the way ovarian cancer is treated,” said Jonathan F. Arambula, vice-president of Research at OncoTEX, in the press release. “The partnership with Sterling will allow us to make OxaliTEX that meets [FDA] regulatory requirements necessary for clinical use.”

“Our mission is to find, fund, and develop bioscience discoveries into life-changing medical innovations, and deliver them to the people who need them the most. Today’s announcement marks a momentous milestone for The iQ Group Global and our novel cancer-fighting drug, OxaliTEX. It brings us closer to our goal of giving the hundreds of thousands of women who are diagnosed with ovarian cancer each year access to a powerful new weapon,” said George Syrmalis, CEO and chairman of The iQ Group Global, in the press release.

“The challenges involved in the manufacture of this complex molecule was a perfect fit for the expertise and capabilities that our team in Germantown have,” added Andrew Henderson, chief commercial officer at Sterling Pharma Solutions, in the press release. “We have worked closely with the OncoTEX team to identify a suitable development and manufacturing strategy, and we look forward to continuing our partnership with them throughout the process to ensure the greatest chance of advancing this new therapy through clinical development.”

Sterling Pharma Solutions and OncoTEX have entered into a clinical development agreement for the manufacturing of OncoTex’s novel platinum-resistant oncology drug candidate, OxaliTEX, for use in clinical trials.

Sterling Pharma Solutions and OncoTEX to Develop Novel Treatment Against Ovarian Cancer

Syngene announced on April 26, 2021 that it has extended its research collaboration with Bristol Myers Squibb through the end of 2030.

The collaboration, which has been in place since 1998, will focus on drug discovery including chemistry, biology, drug metabolism, and pharmacokinetics, along with translational medicine research and pharmaceutical development, including chemical process development and analytical sciences, Sygene said in a company press release. The partnership extension will also include a 40% increase in the number of scientists and the addition of a new 50,000-ft2 dedicated laboratory space.

"For more than 20 years we have considered our work with Bristol Myers Squibb to set the benchmark for integrated research collaborations and are delighted with our joint commitment to extend this collaboration until at least the end of the decade, as well as expand our focus to new areas of science,” said Jonathan Hunt, MD, CEO, Syngene, in the press release. “I have every confidence that together we will continue to deliver science that will improve the lives of patients around the world."

"We greatly value our collaboration with Syngene International and are proud to extend and expand our work together,” added Gregory Vite, PhD, senior vice-president, Small Molecule Drug Discovery, Bristol Myers Squibb, in the press release. “The dedicated facilities and scientific team at Syngene play an important role in helping us realize our vision to transform patients’ lives through science.”

The collaboration, which has been in place since 1998, will focus on drug discovery, translational medicine research, and pharmaceutical development.