Pharmaceutical Technology Editors

FDA has published its review of the accomplishments the agency achieved in 2021. 

FDA 2021 Year in Review–Working for You 

details the agency’s response to COVID-19, including vaccine, treatment, and testing authorizations. FDA has also been monitoring variants of the virus and issuing guidance and contingency plans for dealing with the pandemic. The report also discusses the agency’s efforts for addressing the misuse of prescription drugs, the problem of fraudulent medicines, and the advancement of generic drugs and biosimilars, as well as its work in food safety and tobacco products.

“While much of the attention the agency received in 2021 focused on our response to the pandemic, I am pleased to note that, despite a great deal of uncertainty, the agency was able to accomplish a wide range of its priorities that are already protecting the health and well-being of millions of people living in the US,” said Acting Commissioner Janet Woodcock, MD, in the report. “Every year the FDA reviews hundreds of product applications, ultimately determining which drugs, devices, or biological products (e.g., vaccines) will be marketed in the US. The process involved is rigorous, thoughtful and always adheres to standards for safety and effectiveness. We help to ensure that the human and animal food supply is safe, sanitary, and accurately labeled, and that cosmetic products are safe and properly labeled,” said Woodcock.

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The agency discusses its response to COVID-19 and other milestones in its year-end review.

FDA Publishes 2021 Year in Review

On January 18, 2022, the European Medicines Agency (EMA) stated that its COVID-19 task force reviewed several studies involving approximately 65,000 pregnancies and did not find any sign of increased risk of complications, miscarriages, preterm births, or adverse events after vaccination with mRNA vaccines. “Despite some limitations in the data, the results appear consistent across studies looking at these outcomes,” the agency stated in a 

According to EMA, the studies also showed the vaccines reduce the risk of hospitalization and death in pregnant people as well as non-pregnant people, and side effects related to the mRNA vaccines in pregnant people matched those of non-pregnant people. “Given that so far pregnancy has been associated with a higher risk of severe COVID-19 particularly in the second and third trimesters, people who are pregnant or might become pregnant in the near future are encouraged to get vaccinated in line with national recommendations,” EMA stated in the release.

Because clinical trials do not usually include pregnant people, according to EMA, data on the use of the vaccines are obtained after authorization. The agency will review the latest data on these vaccines administered during pregnancy and will update product information as necessary. “In line with the EU’s safety monitoring plan for COVID-19 vaccines, those vaccines are closely monitored, and relevant new information emerging is continuously collected and promptly reviewed. Although very large numbers of people have already received COVID-19 vaccines, certain side effects may still emerge as more and more people are included in vaccination programmes. EMA’s safety committee, 

 will continue to monitor safety in pregnancy,” the agency stated.

The agency’s COVID-19 task force says latest data shows use of COVID-19 mRNA vaccines during pregnancy is safe.

COVID-19 Vaccines Safe for Use During Pregnancy, EMA States

Prellis Biologics, a US-based biotechnology company, announced on Jan. 13, 2022 that it has entered into a multi-target drug discovery collaboration and licensing agreement with Bristol Myers Squibb, which will utilize Prellis’ externalized human immune system (EXIS) to create high-affinity human antibodies targeting human proteins.

Under the agreement, Prellis and Bristol Myers Squibb will generate human antibody libraries for select targets. Bristol Myers Squibb will pay Prellis an undisclosed up-front payment and funding for research activities as well as development, sales milestones, and royalties for licensed antibodies.

Prellis’ EXIS platform is based on human lymph node organoids, which can break tolerance mechanisms 

allowing for the creation of high affinity human antibodies.

"…[T]he EXIS platform provides high affinity antibodies, derived from lymph node organoids printed with Prellis’ foundational technology, using human PBMCs [peripheral blood mononuclear cells]," said Melanie P. Matheu, founder and CEO of Prellis, in a company press release. "We are looking forward to working with Bristol Myers Squibb to demonstrate advantages of 

 human antibody discovery with the EXIS platform."

"The EXIS platform effectively breaks tolerance, enabling direct discovery of human antibodies to challenging human antigens in less than two week," said Kevin Chapman, chief scientific officer of Prellis, in the press release.

In their collaboration, Bristol Myers Squibb and Prellis Biologics aim to generate human antibody libraries for select targets to enable drug discovery.

Bristol Myers Squibb and Prellis Biologics Partner to Build Human Antibody Libraries

Following their July 2021 merger, Protagen Protein Services, BioAnalytix, and GeneWerk announced on Jan. 12, 2022 that their unified operations and re-branded name is ProtaGene, a contract research organization for the biopharmaceutical and cell and gene therapy industries.

The three legacy companies' combined synergy provides advanced, integrated, and complete protein and gene analytic services for biological therapeutics and cell and gene therapy platforms. ProtaGene provides services from discovery to commercialization.

Previously, Protagen Protein Services and BioAnalytix merged in April 2019, and GeneWerk, which specializes in vector safety, integration site analysis, and bioinformatics, was combined with the group in 2021.

"We are very excited to launch ProtaGene. The track record and reputation of the three legacy entities are individually impressive, and their combination into a single, globally integrated business delivers significant compounded value for our sponsors," said Martin Blüggel, CEO of ProtaGene and the founder of Protagen Protein Services, in a company press release.

"By combining our capabilities, platforms and knowledge, we can now provide our US-based biopharmaceutical and gene therapy customers advanced access to the broadest analytical offering from discovery to market, from a single vendor relationship," said Kirtland Poss, CEO-US of ProtaGene and the founder of BioAnalytix, in the press release.

"Our unique experience in gene analytics—especially integration site analysis, vector purity, vector copy numbers, and bioinformatics analysis for preclinical development and clinical trials—will support many innovators in taking breakthrough therapies to the clinic," added Annette Deichmann, senior vice-president of Cell and Gene Therapy at ProtaGene and the co-founder of GeneWerk, in the press release.

After a previously announced merger, Protagen Protein Services, BioAnalytix, and GeneWerk have formed a new CRO under the name ProtaGene.

Protagen Protein Services, BioAnalytix, and GeneWerk Combine to Form New CRO, ProtaGene 

Pfizer and Beam Therapeutics, a biotechnology company developing precision genetic medicines through base editing, announced an exclusive four-year research collaboration on Jan. 10, 2022. The collaboration, worth up to $1.35 billion, will use Beam’s 

 base editing programs to conduct research on three undisclosed targets for rare genetic diseases of the liver, muscle, and central nervous system.

The base editing program will leverage Beam’s proprietary

 delivery technologies, which use messenger RNA (mRNA) and lipid nanoparticles (LNP) to deliver base editors to target organs. These technologies are designed to enable precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA. According to a Pfizer press release, this would create an efficient edit when compared to traditional gene editing methods.

“At Pfizer, we believe in the powerful potential of mRNA and LNP technologies to address the greatest unmet needs for patients, as evidenced by the beneficial impact our mRNA/LNP-based COVID-19 vaccine is having on the pandemic,” said Mikael Dolsten, chief scientific officer and president, Worldwide Research, Development and Medical, Pfizer, in the press release. “We have a strong history in developing gene replacement therapies for rare diseases, and we see this collaboration with Beam as an opportunity to advance the next generation of gene editing therapies – an exciting scientific frontier – potentially leading to transformation for people living with rare genetic diseases.”

“We are thrilled to partner with Pfizer, a global leader in the design, development, and commercialization of novel medicines,” said John Evans, CEO of Beam, in the press release. “Our leading platform for precision genetic medicine has greatly evolved over the last few years, and we are committed to ensuring the broadest reach of these potentially life-changing technologies. This collaboration will provide a unique opportunity to create potentially transformative base editing programs for indications with critical unmet needs, leveraging our proprietary base editing technology and expanding delivery capabilities. We look forward to working together with Pfizer to advance these technologies and potentially expand our impact for people suffering from serious diseases.”

Pfizer will pay Beam $300 million upfront for their services in this collaboration. Should Pfizer exercise its opt-in license rights for all three targets, they will be eligible for development, regulatory, and commercial milestone payments for potential total deal consideration of up to $1.35 billion, in addition to royalties on global net sales.

Pfizer will pay Beam $300 million upfront to conduct research on three undisclosed base editing targets in the liver, muscle, and central nervous system.

Pfizer and Beam Enter Exclusive Multi-Target Research Collaboration

Forge Biologics, a gene-therapy focused contract development and manufacturing organization, announced plans to accelerate expansion plans for their manufacturing facility in Columbus, Ohio on Jan. 10, 2022. The $240 million expansion is set to increase the overall manufacturing footprint of the company to over 200,000 ft².

According to a company press release, the expansion will significantly increase the number and capacity of Forge’s bioreactors, including installation of 5000-L bioreactors for large-scale current good manufacturing practice (CGMP) client needs. The company expects these to be available for client use in the latter half of 2022.

The expansion will also include 20 CGMP manufacturing suites, with the ability to add more suites in the current facility in the future. Additional automated CGMP fill/finish capabilities, designed with a cleanroom background classifications that meet European Medicines Agency requirements for late-stage clinical and commercial filling needs, will be included as well.

“We are excited to deliver on our goal of offering our clients expanded manufacturing capacity, while providing access to some of the largest suspension bioreactors available in the industry,” said Timothy J. Miller, CEO, president, and co-founder of Forge, in the press release. “This increased scale, combined with our automated fill/finish capabilities, positions Forge as an ideal partner for clients moving beyond the clinic to commercial launch and supply, particularly those needing to manufacture CGMP [adeno-associated virus therapies.]”

Forge Biologics secured the funding for the accelerated expansion through an $80 million secured financing agreement with MidCap Financial. The expansion, which was initially announced in 2021, had secured $160 million in funding prior to this agreement.

CDMO Forge Biologics will accelerate its manufacturing capacity expansion using an additional $80 million in funding it recently secured.