Immune Response and Packaging - Drug Discovery Digest

Case study: gget’s new Open Target module

The Open Targets Blog

JANUARY 30, 2025

gget ( [link] ) is a free, open-source command-line tool and Python package designed to enable efficient querying of large genomic databases, such as Ensembl, UniProt, and NCBI. We chatted to the team about the module, and how it facilitates access to Open Targets Platform data. What is gget?

Bioinformatics

Bioinformatics Immune Response RNA Packaging

Unveiling neoantigen-directed cancer treatment

Drug Target Review

JUNE 9, 2023

Within the collaboration, Adaptive is responsible for end-to-end preclinical TCR discovery. As part of the TCR discovery process and preclinical data package, Adaptive performs rigorous functional characterisation and safety assessments of potent, therapeutic grade antigen-specific TCRs.

Treatment

Treatment Packaging Clinical Research Therapies

Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

OCTOBER 12, 2023

Can they deliver healing genes without triggering an overactive immune response? The details are disturbingly reminiscent of the famous case of Jesse Gelsinger , who died from a ferocious immune response to experimental gene therapy in September 1999. An immune response is two-tiered.

Therapies

Therapies Immune Response DNA Virus

A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

JUNE 27, 2024

The first problem they had to tackle was the editor’s size, because the editor needs to be small enough to be packaged and delivered to specific cells in the body. ZFPs are also common in human cells, meaning they are less likely to trigger an immune response against themselves than the bacterial Cas9.

Disease

Disease Therapies Clinical Trials Engineering

Months of Work Hang in the Balance as Moderna’s COVID-19 Vaccine Awaits AdComm Vote

The Pharma Data

DECEMBER 16, 2020

Gustave Perna, who oversees the logistics for Operation Warp Speed, told reporters earlier this week that McKesson will package and distribute the vaccine to more than 3,000 sites across the country. Instead, it uses a genetic code, which tricks the body into developing an immune response to the virus.

Vaccine

Vaccine Immune Response FDA FDA Approval

FDA Returns Disappointing News for ALS Stem Cell Therapy

PLOS: DNA Science

OCTOBER 19, 2023

If MSCs come from the patient (autologous), then an immune response shouldn’t be a hindrance. Might MSCs introduced into a patient’s spinal fluid somehow “know” to divide and develop into the needed motor neurons? Or secrete neurotrophic factors right where they’re needed?

Therapies

Therapies FDA Clinical Trials Disease

Novavax COVID-19 vaccine demonstrates 90% overall efficacy and 100% protection against moderate and severe disease in PREVENT-19 Phase 3 trial

The Pharma Data

JUNE 25, 2021

NVX-CoV2373 was created using Novavax’ recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is formulated with Novavax’ patented saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies. efficacy against the B.1.351

Vaccine

Vaccine Trials Disease Immune Response

New insights into the role of viral capsids in gene therapy safety

Drug Target Review

DECEMBER 5, 2024

2 However, utilisation of biological systems introduces variability and by-products, as well as a challenge unique to AAV production: the assembly of full capsids with correctly packaged DNA, partially filled capsids containing partial vector and/or host cell DNA, and empty capsids.

Therapies

Therapies Virus DNA Compliance

Regulatory Submissions for Dengue Vaccine Candidate in EU and Dengue-Endemic Countries

The Pharma Data

MARCH 25, 2021

2 Clinical Phase 2 data in children and adolescents showed that TAK-003 induced immune responses against all four dengue serotypes, in both seropositive and seronegative participants, which persisted through 48 months after vaccination, and the vaccine was found to be generally safe and well tolerated.

Vaccine

Vaccine Virus Trials Hospitals

Codon Digest: Seeing Colors After Gene Therapy

Codon

JULY 27, 2023

A new paper from Michael Elowitz’s group at Caltech reports engineered RNA exporters that “efficiently and specifically package and secrete target RNA molecules from mammalian cells within protective nanoparticles.” a lower risk of immune responses. No longer. ” And a third paper (BOOM!)

Therapies

Therapies RNA DNA Engineering

Codon Digest: Seeing Colors After Gene Therapy

Codon

JULY 27, 2023

A new paper from Michael Elowitz’s group at Caltech reports engineered RNA exporters that “efficiently and specifically package and secrete target RNA molecules from mammalian cells within protective nanoparticles.” a lower risk of immune responses. No longer. ” And a third paper (BOOM!)

Therapies

Therapies RNA DNA Engineering

Regeneron Announces Encouraging Initial Data from COVID-19 Antibody Cocktail Trial in Hospitalized Patients on Low-flow Oxygen

The Pharma Data

DECEMBER 29, 2020

The analysis was prospectively designed to focus on patients who had not yet mounted their own immune response to SARS-CoV-2 (i.e., did not have antibodies at baseline: seronegative), as prior evidence suggested these patients were at greater risk. futility analysis). .

Hospitals

Hospitals Trials Clinical Trials Production

Tonix Pharmaceuticals Enrolls First Participant in the PRECISION Study, an Observational Study to Facilitate Development of Precision Medicine Techniques for COVID-19 Vaccines and Therapeutics

The Pharma Data

OCTOBER 14, 2020

(Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that the first participant was enrolled in the observational PRECISION study (TNX-C002), to examine the immune responses to COVID-19 in healthy volunteers who have recovered from COVID-19 or were asymptomatic.

Vaccine

Vaccine Pharmaceuticals Small Molecule Virus

Regeneron’s Casirivimab and Imdevimab Antibody Cocktail for COVID-19 is First Combination Therapy to Receive FDA Emergency Use Authorization

The Pharma Data

NOVEMBER 20, 2020

The clinical evidence from Regeneron’s outpatient trial suggests that monoclonal antibodies such as casirivimab and imdevimab have the greatest benefit when given early after diagnosis and in patients who have not yet mounted their own immune response or who have high viral load.

Therapies

Therapies FDA Clinical Trials Hospitals

Regeneron’s COVID-19 Outpatient Trial Prospectively Demonstrates that REGN-COV2 Antibody Cocktail Significantly Reduced Virus Levels and Need for Further Medical Attention

The Pharma Data

OCTOBER 28, 2020

“We continue to see the strongest effects in patients who are most at risk for poor outcomes due to high viral load, ineffective antibody immune response at baseline, or pre-existing risk factors. Yancopoulos , M.D., President and Chief Scientific Officer of Regeneron. Regeneron has shared these results with the U.S.

Virus

Virus Trials Clinical Trials Production

Scaling Phage Therapy

Codon

FEBRUARY 13, 2024

Such contaminants could have the same toxic effects as the bacterial infection, or provoke a severe immune response in the patient, and therefore the phage mixtures must go through extensive purification and testing procedures. I cover this more extensively in The Good Virus.

Therapies

Therapies Virus DNA Clinical Trials

New England Journal of Medicine Publishes Positive Initial Regeneron Antibody Cocktail Results in Non-hospitalized Patients with COVID-19

The Pharma Data

DECEMBER 17, 2020

Senior Vice President and Head of Global Clinical Development at Regeneron and lead author of the publication. “The investigational cocktail is now available to indicated high-risk U.S. patients under an Emergency Use Authorization, and we also continue a robust clinical development program.”

Hospitals

Hospitals Production Clinical Trials Virus

Regeneron’s REGEN-COV2 is First Antibody Cocktail for COVID-19 to Receive FDA Emergency Use Authorization

The Pharma Data

NOVEMBER 21, 2020

The clinical evidence from Regeneron’s outpatient trial suggests that monoclonal antibodies such as REGEN-COV2 have the greatest benefit when given early after diagnosis and in patients who have not yet mounted their own immune response or who have high viral load.

FDA

FDA Clinical Trials Hospitals Production

Synthetic Origins

Codon

APRIL 2, 2024

Like, if we start thinking about therapeutic circuits, how can we design the components in such a way that they are not going to cause an immune response? So we engineered a system that allows the cell to package up RNAs and then secrete them in extracellular vesicles. Another one is immunogenicity.

RNA

RNA Engineering Molecular Biology Synthetic Chemistry

Lilly Delivers Strong Second-Quarter 2021 Financial Results, Updates 2021 Financial Guidance

The Pharma Data

AUGUST 3, 2021

The company confirmed the tirzepatide SURPASS program has met global regulatory submission requirements for evaluating cardiovascular risk and its intention to submit the registration package to regulatory authorities by the end of 2021. Loxo Oncology at Lilly and Kumquat Biosciences Inc.

Production

Production Marketing Research DNA

Article FDA Thank You Draft guidance on potency assays for CGT products garners extensive stakeholder input

Agency IQ

APRIL 12, 2024

For therapeutic protein products , a critical use of assays is the detection and assessment of potential anti-drug antibody (ADA) formation, signaling a product’s potential to induce an unwanted immune response.

Production

Production FDA Therapies Biochemical Assays

Biden Warns of 500,000 COVID Deaths by February

The Pharma Data

JANUARY 22, 2021

trillion coronavirus aid package, will need the cooperation of Congress to carry out much of his ambitious plan, which also includes greatly expanding testing of asymptomatic people to reopen schools and businesses, the Times said. . But the president, who has proposed a $1.9

Vaccine

Vaccine Immune Response Packaging Disease

America Sees COVID Deaths Top 400,000, While New Variants Worry Scientists

The Pharma Data

JANUARY 20, 2021

“This is the first time I’ve been concerned about a variant partially evading the immune response and partially evading the vaccine.” trillion pandemic “rescue” package that includes $20 billion for the vaccine effort. ” Both experts stressed that people should still get the vaccine.

Vaccine

Vaccine Immune Response Virus Pharmacy

Evusheld long-acting antibody combination recommended for approval in the EU for the pre-exposure prophylaxis (prevention) of COVID-19

The Pharma Data

MARCH 25, 2022

AstraZeneca’s Evusheld (tixagevimab co-packaged with cilgavimab), a long-acting antibody combination, has been recommended for marketing authorisation in the European Union (EU) for the pre-exposure prophylaxis (prevention) of COVID-19 in a broad population of adults and adolescents aged 12 years and older weighing at least 40 kg.

Vaccine

Vaccine Virus Trials Immune Response

Evusheld long-acting antibody combination approved in the EU for pre-exposure prophylaxis (prevention) of COVID-19 in a broad population

The Pharma Data

MARCH 29, 2022

AstraZeneca’s Evusheld (tixagevimab co-packaged with cilgavimab), a long-acting antibody combination, has been granted marketing authorisation in the European Union (EU) for the pre-exposure prophylaxis (prevention) of COVID-19 in a broad population of adults and adolescents aged 12 years and older weighing at least 40 kg.

Vaccine

Vaccine Virus Trials Immune Response

The Forgotten Pandemic

Codon

OCTOBER 27, 2024

Interferon gamma release assays pick up an immune response to the infection. tuberculosis in his system, his T cells will recognize the antigen and mount an intense immune response at the injection site, leaving behind a red and swollen bull’s-eye. A sputum test stain for M. tuberculosis. Should Enzokuhle have M.

Immune Response

Immune Response Doctors Disease Laboratories

Codon Digest: Ghana Approves A Better Malaria Vaccine

Codon

APRIL 23, 2023

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain.

Vaccine

Vaccine DNA Virus Engineering

Codon Digest: Ghana Approves A Better Malaria Vaccine

Codon

APRIL 23, 2023

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Unfortunately, they can also trigger immune responses, and they are not super efficient at gene-editing some parts of the brain.

Vaccine

Vaccine DNA Virus Engineering

Biden Unveils Details of National Pandemic Response Plan

The Pharma Data

JANUARY 21, 2021

billion coronavirus aid package, will need the cooperation of Congress to carry out much of his ambitious plan, which also includes greatly expanding testing of asymptomatic people to reopen schools and businesses, the Times said. . But the president, who has proposed a $1.9

Vaccine

Vaccine Immune Response Packaging Production

Drug Discovery Digest

Case study: gget’s new Open Target module

Unveiling neoantigen-directed cancer treatment

Trending Sources

Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

A therapy candidate for fatal prion diseases turns off disease-causing gene

Months of Work Hang in the Balance as Moderna’s COVID-19 Vaccine Awaits AdComm Vote

FDA Returns Disappointing News for ALS Stem Cell Therapy

Novavax COVID-19 vaccine demonstrates 90% overall efficacy and 100% protection against moderate and severe disease in PREVENT-19 Phase 3 trial

New insights into the role of viral capsids in gene therapy safety

Regulatory Submissions for Dengue Vaccine Candidate in EU and Dengue-Endemic Countries

Codon Digest: Seeing Colors After Gene Therapy

Codon Digest: Seeing Colors After Gene Therapy

Regeneron Announces Encouraging Initial Data from COVID-19 Antibody Cocktail Trial in Hospitalized Patients on Low-flow Oxygen

Tonix Pharmaceuticals Enrolls First Participant in the PRECISION Study, an Observational Study to Facilitate Development of Precision Medicine Techniques for COVID-19 Vaccines and Therapeutics

Regeneron’s Casirivimab and Imdevimab Antibody Cocktail for COVID-19 is First Combination Therapy to Receive FDA Emergency Use Authorization

Regeneron’s COVID-19 Outpatient Trial Prospectively Demonstrates that REGN-COV2 Antibody Cocktail Significantly Reduced Virus Levels and Need for Further Medical Attention

Scaling Phage Therapy

New England Journal of Medicine Publishes Positive Initial Regeneron Antibody Cocktail Results in Non-hospitalized Patients with COVID-19

Regeneron’s REGEN-COV2 is First Antibody Cocktail for COVID-19 to Receive FDA Emergency Use Authorization

Synthetic Origins

Lilly Delivers Strong Second-Quarter 2021 Financial Results, Updates 2021 Financial Guidance

Article FDA Thank You Draft guidance on potency assays for CGT products garners extensive stakeholder input

Biden Warns of 500,000 COVID Deaths by February

America Sees COVID Deaths Top 400,000, While New Variants Worry Scientists

Evusheld long-acting antibody combination recommended for approval in the EU for the pre-exposure prophylaxis (prevention) of COVID-19

Evusheld long-acting antibody combination approved in the EU for pre-exposure prophylaxis (prevention) of COVID-19 in a broad population

The Forgotten Pandemic

Codon Digest: Ghana Approves A Better Malaria Vaccine

Codon Digest: Ghana Approves A Better Malaria Vaccine

Biden Unveils Details of National Pandemic Response Plan

Stay Connected