Containment, FDA Approval, Gene and Immune Response

Qalsody (Tofersen) Becomes First-of-Its-Kind Treatment for ALS

XTalks

MAY 1, 2023

Last week, the US Food and Drug Administration (FDA) granted accelerated approval to Biogen’s Qalsody (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 ( SOD1 ) gene. How Does Qalsody Work?

FDA Approval

FDA Approval Gene Genetics Protein

Biologic Therapeutics Development, Part 2: Regulatory Pathways and Pharmacometric Analysis

Camargo

DECEMBER 13, 2021

Both the FDA’s Center for Drug Evaluation and Research (CDER) and its Center for Biologics Evaluation and Research (CBER) have regulatory responsibility for therapeutic biological products, which are subject to both the Federal Food, Drug and Cosmetic (FD & C) Act and the Public Health Service (PHS) Act.

Development

Development Production Immune Response Licensing

The democratisation of cell and gene therapy

Drug Discovery World

MAY 29, 2024

Marc Hummersone, Senior Director of Research and Development (R&D) at Astrea Bioseparations, shares insight on the challenges and opportunities in cell and gene therapy (CGT) with DDW’s Megan Thomas. But the unpacked capsid can give rise to an immune response, which is not good for the patient.

Gene Therapy

Gene Therapy Gene Genetics DNA

Malaria Vaccine a Breakthrough Success with 77 Percent Efficacy

XTalks

APRIL 26, 2021

Related: After 18 Years, FDA Approves Malaria Prevention Drug. This is because there are thousands of genes in malaria parasites compared to only about a dozen or so in coronaviruses. Moreover, a very strong immune response is needed to fend off the disease. Malaria Vaccine Phase II Trial. At six months, 29.5

Vaccination

Vaccination Vaccine Immune Response Trials

Understanding the microbiome as an ‘overlooked critical organ’

pharmaphorum

APRIL 26, 2022

The microbiome, as a term, can be related to the nasal, oral, skin, gastro-intestinal, and urogenital regions of the human body, each containing varied microbial communities that have an impact on health. If approved by the FDA, the treatment would become the first microbiome-based treatment to receive approval.

Containment

Containment Production Development Immune Response

The path to improved safety of gene-based products

Drug Discovery World

MAY 11, 2023

Kerstin Pohl , Senior Global Marketing Manager, Gene Therapy & Nucleic Acid at SCIEX, looks at the application of liquid chromatography-mass spectrometry for analysing host cell proteins. Gene-based therapeutics and vaccines hold immense potential for more efficient and personalised treatment and prevention of complex diseases.

Production

Production Gene Protein Vaccination

FDA Authorizes Phase 1 Trial of ImmunityBio’s Novel COVID-19 Vaccine Candidate hAd5; Dual Construct is Designed to Drive Both T Cell and Antibody Immunity

The Pharma Data

OCTOBER 14, 2020

Additionally, immunogenicity, duration of immune response and occurrence of symptomatic COVID-19 will be measured. This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Cancer Gene Ther. Forward-Looking Statements. iv Osada T, et al.

Antibody

Antibody Vaccination Vaccine Trials

Lineage Cell Therapeutics Provides End of Year Shareholder Letter

The Pharma Data

DECEMBER 27, 2020

Notably, we never modify the DNA of these cells, which avoids some of the safety concerns that have been reported with certain gene-editing technologies. Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-Looking Statements.

Manufacturing

Manufacturing Production Development Clinical Trials

Using bispecific antibodies to advance cancer immunotherapy

pharmaphorum

NOVEMBER 2, 2021

The more targeted, bispecific functionality of bsAbs broadens the reach of antibody-mediated therapies against both solid and haematological cancers, creating novel approaches that can induce a tumour-specific immune response, target immune checkpoints, or improve payload delivery to tumour cells (Fig.

Antibody

Antibody Immune Response Production Engineer

Genentech’s Novel Anti-TIGIT Tiragolumab Granted FDA Breakthrough Therapy Designation in Combination With Tecentriq for PD-L1-High Non-Small Cell Lung Cancer

The Pharma Data

JANUARY 4, 2021

Tiragolumab is the first anti-TIGIT molecule to be granted BTD from the FDA, and the designation is based on randomized data from the Phase II CITYSCAPE trial. CITYSCAPE provides the first evidence that targeting both immune inhibitory receptors, TIGIT and PD-L1, may enhance anti-tumor activity by potentially amplifying the immune response.

Immune Response

Immune Response Medicine Protein Gene

Transgene Announces Detailed Results From Clinical Study of TG4001 in Combination With Avelumab in Advanced HPV-positive Cancers

The Pharma Data

OCTOBER 26, 2020

The treatment induced HPV-specific T-cell responses and was associated with increased levels of immune cell infiltration in the tumors and expression of genes associated with activation of the immune system. An overall response rate of 23.5% . – .

Vaccination

Vaccination Vaccine Trials Containment

Sanofi continued its growth trajectory. Strong increase in Q1 2021 business EPS(1) at CER

The Pharma Data

APRIL 27, 2021

At the end of March, the FDA approved Sarclisa ® in combination with carfilzomib and dexamethasone for patients with relapsed multiple myeloma. Developed as part of a joint-partnership between Sanofi and Merck, Vaxelis ® is the first and only hexavalent combination vaccine approved in the U.S. First-quarter sales in the U.S.

Sales

Sales Vaccination Vaccine Medicine

CheckMate -577, a Phase 3 Trial Evaluating Opdivo (nivolumab) as Adjuvant Therapy for Patients with Resected Esophageal or Gastroesophageal Junction Cancer, Meets Primary Endpoint of Disease-Free Survival

The Pharma Data

AUGUST 11, 2020

In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.

Trials

Trials Hormones Clinical Trials Clinical Trials

CheckMate -649, a Phase 3 Trial Evaluating Opdivo (nivolumab) Plus Chemotherapy vs. Chemotherapy, Meets Primary Endpoints Demonstrating Superior Overall Survival and Progression-Free Survival in First-Line Treatment of Gastric and Esophageal Cancers

The Pharma Data

AUGUST 11, 2020

In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.

Trials

Trials Hormones Clinical Trials Clinical Trials

Regeneron’s Inmazeb named Galien’s Best Biotech Product

pharmaphorum

OCTOBER 31, 2022

It is the first FDA-approved treatment for Zaire ebolavirus. Yancopoulos continued: “Our scientists realised that the science demanded they adapt these technologies to deliver the first FDA-approved recombinant monoclonal antibody treatment for any viral disease, and they picked one of the most challenging ones in Ebola.”.

Production

Production FDA Approval Antibody Gene

Clinical Research Informer

Qalsody (Tofersen) Becomes First-of-Its-Kind Treatment for ALS

Biologic Therapeutics Development, Part 2: Regulatory Pathways and Pharmacometric Analysis

Trending Sources

The democratisation of cell and gene therapy

Malaria Vaccine a Breakthrough Success with 77 Percent Efficacy

Understanding the microbiome as an ‘overlooked critical organ’

The path to improved safety of gene-based products

FDA Authorizes Phase 1 Trial of ImmunityBio’s Novel COVID-19 Vaccine Candidate hAd5; Dual Construct is Designed to Drive Both T Cell and Antibody Immunity

Lineage Cell Therapeutics Provides End of Year Shareholder Letter

Using bispecific antibodies to advance cancer immunotherapy

Genentech’s Novel Anti-TIGIT Tiragolumab Granted FDA Breakthrough Therapy Designation in Combination With Tecentriq for PD-L1-High Non-Small Cell Lung Cancer

Transgene Announces Detailed Results From Clinical Study of TG4001 in Combination With Avelumab in Advanced HPV-positive Cancers

Sanofi continued its growth trajectory. Strong increase in Q1 2021 business EPS(1) at CER

CheckMate -577, a Phase 3 Trial Evaluating Opdivo (nivolumab) as Adjuvant Therapy for Patients with Resected Esophageal or Gastroesophageal Junction Cancer, Meets Primary Endpoint of Disease-Free Survival

CheckMate -649, a Phase 3 Trial Evaluating Opdivo (nivolumab) Plus Chemotherapy vs. Chemotherapy, Meets Primary Endpoints Demonstrating Superior Overall Survival and Progression-Free Survival in First-Line Treatment of Gastric and Esophageal Cancers

Regeneron’s Inmazeb named Galien’s Best Biotech Product

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