The Pharma Data

SEPTEMBER 30, 2021

Anderson Professor of Chemical and Biomolecular Engineering, and his colleagues, are reporting in iScience the event of an intranasal subunit vaccine that gives durable local immunity against inhaled pathogens. A fundamental limitation of intramuscular vaccines is that they’re not designed to elicit mucosal immunity.

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Vial

DECEMBER 11, 2023

Pathophysiological evidence links HCV infections to the risk of liver diseases such as hepatocellular carcinoma (HCC) and liver cirrhosis. Patients at advanced stages of liver disease can still be treated. To what extent does viral eradication reduce the risk of liver disease progression in the long term?

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The Pharma Data

APRIL 26, 2022

Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, and Pfizer Inc. Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, and Pfizer Inc. NYSE: PFE) today reported positive Phase 2 pediatric data for their Lyme disease vaccine candidate, VLA15.

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Drug Target Review

MAY 22, 2024

How does Circio’s circVec technology aim to enhance the potency and reduce the cost of current gold-standard gene therapy? As a first step, Circio is working on designing and validating circVec-AAV vectors for the treatment of genetic diseases, and testing these head-to-head against the mRNA-AAV equivalent in vivo.

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The Pharma Data

JULY 21, 2021

Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company focused on the development and commercialization of prophylactic vaccines for infectious diseases with significant unmet medical need, and Pfizer Inc. at Month 7). Juan Carlos Jaramillo M.D., About VLA15.

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Drug Target Review

MARCH 21, 2024

Dystrophin is mutated in Duchenne muscular dystrophy (DMD), a rare disease that causes progressive muscle weakness and atrophy. There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. Of note, mRNA is redosable as opposed to AAV.

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Codon

APRIL 30, 2023

Also, last week’s digest had an error: The country that approved the malaria vaccine was Ghana. Vaccines (basically little fat bubbles filled with mRNA and some dissolvable polymers) were physically printed onto microneedle patches using a robot. The patches have little spikes that help push the vaccines through the skin.

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Codon

APRIL 30, 2023

Also, last week’s digest had an error: The country that approved the malaria vaccine was Ghana. Vaccines (basically little fat bubbles filled with mRNA and some dissolvable polymers) were physically printed onto microneedle patches using a robot. The patches have little spikes that help push the vaccines through the skin.

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Codon

NOVEMBER 3, 2024

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. Even after microbiologists discovered the bacterium that causes the illness in 1882, it wasn’t until the 1920s that researchers were able to develop a vaccine for TB.

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Now, FDA’s CBER is setting its sights on making ambitious strides toward tackling rare diseases.

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PPD

OCTOBER 24, 2024

Coordinated efforts are needed to combat this disease and improve the clinical status of persons with HBV infection. The new guidelines emphasize the importance of early diagnosis and treatment to reduce the disease burden and improve patient outcomes.

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The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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Drug Target Review

JANUARY 23, 2024

Vaccinations against tumour antigens that are shared between tumours, or tumour antigens that arise from mutations unique to individual tumours, represent promising strategies. Cell-based therapies, such as tumour-infiltrating lymphocyte (TIL) therapy, have also drawn significant attention following promising results in a melanoma trial.

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The Pharma Data

JUNE 17, 2022

In a Phase 2/3 clinical trial, the Pfizer-BioNTech COVID-19 Vaccine elicited a strong immune response in this age group Three 3-µg doses had favorable safety profile similar to placebo in young children ages 6 months through 4 years in Phase 2/3 clinical trial Pfizer-BioNTech COVID-19 Vaccine now authorized in the U.S. Ugur Sahin, M.D.,

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Codon

APRIL 9, 2023

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. Read Efficacy and safety of a bivalent RSV prefusion F vaccine in older adults. Molecular Therapy. Gene Therapy. npj Vaccines. Support for $5 per month.

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Drug Target Review

MARCH 1, 2024

How significant are the results in terms of advancing TCR-T cell therapy and AMP immunotherapy for the treatment of solid tumours? We have already applied this approach to several important tumour targets where enhancements in TCR-T therapy may lead to important gains in clinical benefit, including mutated KRAS, NY-ESO, and HPV E7 antigens.

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Chemical Biology and Drug Design

NOVEMBER 2, 2023

Abstract Breast cancer is a common and deadly disease, so there is a constant need for research to find efficient targets and therapeutic approaches. Endocrine treatments and HER2-targeted drugs are examples of targeted therapies now being used against these receptors. Different targets for breast cancer therapeutics.

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ASPET

APRIL 5, 2024

Tailoring the membrane coating to immune cell sources imparts unique characteristics to the nanoparticles suitable for broader application in infectious disease. Their capacity to bind both inflammatory signals and virulence factors assembles the most promising sepsis therapies into a singular, pathogen-agnostic therapeutic.

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The Pharma Data

MARCH 1, 2022

Astellas”) today announced that they have entered into an agreement whereby Affinivax has reacquired the exclusive worldwide rights to ASP3772, a novel vaccine candidate targeting Streptococcus pneumoniae. We believe ASP3772 has significant potential to address unmet needs in this important vaccine category.

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The Pharma Data

JUNE 1, 2022

has been appointed Senior Vice President and Head of the Company’s Vaccine Research & Development (R&D) organization effective August 1, succeeding Kathrin U. Annaliesa is a world-class scientist with a track record of delivering both vaccines and therapeutics in pioneering new areas of science and where there is urgent unmet need.

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The Pharma Data

MAY 31, 2022

Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, and Takeda ( TSE:4502/NYSE:TAK ) announced to transfer the marketing authorization in Japan for Moderna’s COVID-19 vaccine Spikevax TM (mRNA-1273) from Takeda to Moderna in Japan as of August 1, 2022. Moderna, Inc., ” Authorized Use.

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The Pharma Data

JUNE 18, 2021

First approval of a conjugate vaccine that helps protect against 20 serotypes responsible for the majority of invasive pneumococcal disease and pneumonia, 1,2,3,4,5,6,7 including seven responsible for 40% of pneumococcal disease cases and deaths in the U.S. NEW YORK–(BUSINESS WIRE)– Pfizer Inc. Jansen, Ph.D.,

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The Pharma Data

SEPTEMBER 30, 2021

As one among the leading global vaccines companies, we still bring options which help protect people’s health round the world,” said Michael Greenberg, MD, MPH, Medical Head, Sanofi Pasteur North America. “As 4,5 Anyone can get meningococcal disease, but teens and young adults are among those that are at the very best risk for the infection.6,7

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The Pharma Data

MARCH 3, 2022

The FDA decision is informed by the results of the Phase 2b proof-of-concept study of RSVpreF (NCT04032093), a global, double-blinded, placebo-controlled study that assessed the safety and immunogenicity of RSVpreF in healthy pregnant women ages 18 through 49 years old, who were vaccinated between 28- and 36-weeks gestation, and their infants.

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The Pharma Data

AUGUST 5, 2021

The acquisition strengthens BioNTech’s cell therapy pipeline by accelerating the individualized solid tumor Neoantigen TCR cell therapy research and development program. It also expands the Company’s cell therapy capabilities and manufacturing footprint in North America, building on its acquisition of Neon Therapeutics in 2020.

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Agency IQ

JUNE 21, 2024

CBER’s Peter Marks offers insights on a new Rare Disease Hub, upcoming accelerated approval guidance This week, PETER MARKS, director of FDA’s Center for Biologics Evaluation and Research (CBER), spoke at several sessions of the Drug Information Association (DIA) annual meeting.

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Broad Institute

AUGUST 7, 2024

Anna Greka ’s research focuses on fundamental mechanisms of cellular dysfunction in genetic diseases with a focus on membrane proteins. She also leads Broad’s rare disease initiative, Ladders to Cures. Pardis Sabeti , a computational geneticist, is a pioneer in pandemic preparedness and infectious disease surveillance.

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The Pharma Data

FEBRUARY 24, 2022

If the EC grants the variation, the decision will be immediately applicable to all 27 EU member states, making booster vaccines available to everyone 12 years and older. . The COVID-19 vaccine booster for individuals 12 through 15 years of age was already granted Emergency Use Authorization by the U.S. and Europe.

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The Pharma Data

AUGUST 7, 2020

Takeda now has a vaccine project in its fight against the COVID-19 pandemic, alongside repurposing existing drugs and developing a plasma-based therapy to treat the deadly disease. biotech’s coronavirus vaccine candidate, NVX-CoV2373, in Japan. RELATED: Novavax’s COVID-19 vaccine looks promising in early data.

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The Pharma Data

JUNE 25, 2021

In the unprecedented context of at least 13 variants circulating within the study population subset assessed at this interim analysis, CVnCoV demonstrated an interim vaccine efficacy of 47% against COVID-19 disease of any severity and did not meet prespecified statistical success criteria. About CVnCoV. About CVnCoV.

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The Pharma Data

AUGUST 7, 2020

New Phase 1/2 on Novavax’s COVID-19 vaccine candidate NVX?CoV2373 CoV2373 has shown that the therapy generated “robust antibody responses” when used with or without the company’s Matrix?M tolerated COVID-19 vaccine with a robust immunogenicity profile,” explained Dr Gregory Glenn, President of Research and Development at Novavax.

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Alta Sciences

SEPTEMBER 27, 2024

This issue also covers classes of immunomodulators, including monoclonal antibodies, CAR-T cells, and vaccines; immunotherapy trials, focusing on complex study designs and diverse patient populations; and bioanalytical methods and assays, including pharmacokinetics and anti-drug antibodies (ADA).

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The Pharma Data

JUNE 1, 2022

The United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A, a rare and life-threatening bleeding disorder, based on data from the pivotal XTEND-1 P hase 3 stud y.

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Drug Target Review

JULY 17, 2024

How does GigaGen’s single-cell discovery and development platform differ from traditional methods of producing polyclonal antibody therapies? Current methods for producing pAb therapies rely on regular plasma donations for their development.

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The Pharma Data

JULY 28, 2021

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for avalglucosidase alfa, a long-term enzyme replacement therapy for the treatment of people with Pompe disease. Avalglucosidase alfa is also currently undergoing review in other countries including the U.S.,

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The Pharma Data

DECEMBER 15, 2020

Food and Drug Administration (FDA) granted Novartis ’ experimental drug iptacopan Breakthrough Therapy designation in paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder. Breakthrough Therapy designation will expedite the development and review of iptacopan for this indication.

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The Pharma Data

DECEMBER 19, 2020

Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines today announced that the U.S. 11 ACIP members voted in favor of the vaccine and 0 members voted against. We look forward to vaccinations of this important population starting this week.”. .–( BUSINESS WIRE )– Moderna, Inc.,

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