Pharmaceutical Technology Editors

The FDA Vaccines and Related Biologics Products Advisory Committee (VBPAC) unanimously voted on Oct. 15, 2021, to recommend an Emergency Use Authorization (EUA) for a booster dose of the Johnson & Johnson (J&J) COVID-19 vaccine for adults aged 18 or older at least two months following initial vaccination. This decision comes just one day after a 

The vote followed findings from two J&J clinical trials. One was the Phase III ENSEMBLE 2 study which evaluated booster doses of the company’s COVID-19 vaccine administered two months after the single-shot. According to a company press release, these findings suggested increased protection (94%) against moderate-to-severe COVID-19 in the United States.

J&J’s other clinical trial was a Phase I/IIa study evaluating a booster dose given six months after the first shot. According to the press release, this demonstrated a nine-fold increase in antibody levels one week after the booster and a 12-fold increase four weeks after the booster.

In addition to the clinical trials, data from a real-world study that was conducted from March to the end of July, and then extended to the end of August 2021, was also supplied to the council. This study showed stable vaccine effectiveness of 76% for COVID-19 related infections and 81% for COVID-19-related hospitalizations. There was no evidence of reduced effectiveness over the study duration of six months, which included the time period in which the Delta variant became dominant in the US. However, sequencing data was not available for analysis.

“Today’s recommendation is based on the totality of evidence, with clinical and real-world data showing that while a single shot offers strong and long-lasting protection against COVID-19, a booster given after the single-dose primary vaccination increases protection, in particular against symptomatic COVID-19,” said Paul Stoffels, vice-chairman of the Executive Committee and chief scientific officer, Johnson & Johnson, in the press release.

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The council voted 19-0 to recommend that FDA authorize booster doses of the J&J COVID-19 vaccine just one day after granting Moderna a similar recommendation.

FDA Advisory Council Unanimously Supports Johnson & Johnson COVID-19 Booster EUA

Moderna announced on Oct. 14, 2021, that FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) unanimously recommended that FDA grant an Emergency Use Authorization (EUA) for a booster dose of the Moderna COVID-19 vaccine (mRNA-1273). In a 19–0 vote, the VRBPAC recommended a 50 ug booster shot should be made available to three populations:

People aged 18 to 64 who are at high risk for severe COVID-19

People aged 18 to 64 whose exposure to COVID-19 puts them at risk for COVID-19 complications or severe illness.

Additionally, this booster dose is to be administered only after at least six months have passed since their original vaccination series.

A recent Phase II study of mRNA-1273 was conducted wherein 344 interested participants were given a 50-ug booster dose six to eight months following their second dose. According to the study, neutralizing antibody titers, particularly against variants of concern, had waned prior to this study. However, following a booster dose, neutralizing titers were raised significantly to the Phase III benchmark level.

“We thank the committee for their review and for their positive recommendation in support of [EUA]. This positive recommendation is supported by data on the 50-µg booster dose of our COVID-19 vaccine, which shows robust antibody responses against the original virus, but also against the Delta variant,” said Stéphane Bancel, CEO of Moderna, in a company press release. “We remain committed to staying ahead of the virus and following the evolving epidemiology of SARS-CoV-2. We look forward to making our booster available to people in the U[nited] S[tates] to help protect themselves against this ongoing public health emergency.”

This recommendation is non-binding but will be taken into consideration as it concerns an EUA. The Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices will also meet to discuss a recommendation on a booster shot.

The council voted 19–0 to recommend that FDA authorize booster doses of the Moderna COVID-19 vaccine for certain populations.

FDA Advisory Council Unanimously Supports Moderna COVID-19 Booster EUA

Poseida Therapeutics, a clinical-stage biopharmaceutical company specializing in cell and gene therapeutics, announced that it has entered into a research collaboration and exclusive license agreement with Takeda Pharmaceutical Company in an Oct. 12, 2021, press release. In this collaboration, Takeda will utilize Poseida's nanoparticle delivery technology and other proprietary genetic engineering platforms for the research and development of up to eight gene therapies.

This collaboration will focus on developing non-viral 

 gene therapy programs, including Poseida's Hemophilia A program. Poseida will lead research activities up to candidate selection, after which Takeda will assume responsibility for further development and commercialization.

The parties will collaborate to initially develop up to six 

gene therapy programs under the terms of the agreement, utilizing Poseida's technology platforms—including piggyBac, Cas-CLOVER, and biodegradable nanoparticle technology as well as other technologies. In addition, Takeda can add two additional programs to the collaboration and would then provide funding for all collaboration program R&D costs. Poseida will receive an upfront payment of $45 million and preclinical milestones and is eligible to receive future clinical development, regulatory, and commercial milestone payments.

"Our technologies offer highly efficient gene delivery, fully integrated non-viral genome insertion, and ultra-precise site-specific gene editing,” said Eric Ostertag, CEO of Poseida, in a company press release. “Together with Takeda, we look forward to developing potential cures for a number of genetic diseases with high unmet need."

"Poseida's differentiated platform technologies show great promise in developing non-viral 

 gene therapies using their novel genetic engineering and delivery technologies that complement our existing collaborations," said Madhu Natarajan, head of Takeda’s rare diseases drug discovery unit, in the press release.

In this collaboration, Takeda will utilize Poseida's biodegradable DNA and RNA nanoparticle delivery technology and other proprietary genetic engineering platforms for the research and development of gene therapies.

Poseida Therapeutics and Takeda Partner to Develop Non-Viral In-Vivo Gene Therapy Programs

MilliporeSigma, the United States and Canadian life science business of Merck KGaA, Darmstadt, Germany, announced on Oct. 12, 2021, the opening of its second Carlsbad, Calif. facility, which expands its footprint as a contract development and manufacturing organization (CDMO). The $110 million, 140,000-square-footfacility will more than double the life science business sector’s existing capacity to support large-scale commercial and industrial manufacturing for viral gene therapy.

This is MilliporeSigma’s second Carlsbad, Calif. facility, both of which will focus on cell and gene therapy—specifically viral vector-based therapies. There are 30 cleanroom suites between the two facilities that support small- to large-scale clinical and commercial production. In addition, MilliporeSigma is one of the first CDMOs granted FDA and European Medicines Agency approval for viral vector manufacturing, according to the company press release.

“Today, most gene therapies target rare diseases, but there is an urgent need to efficiently manufacture these treatments to reach larger patient populations,” said Matthias Heinzel, CEO, Life Science, Merck, in the press release. “As a leader in viral vector manufacturing, this increase in capacity and scale is the next step in enabling our customers to bring new curative treatments to market.”

MilliporeSigma announced the opening of its second Carlsbad, California-based facility, which doubles production capacity to support commercial and industrial manufacturing of viral vectors. 