Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

Pharmaceutical Technology

APRIL 24, 2023

A journey within nanomedicine Most of the initial research into nanomedicine focused on enzyme replacement therapy in the liver for enzymes such as Aspergillus niger amyloglucosidase , says Moein Moghimi, PhD, professor of Pharmaceutics and Nanomedicine, Newcastle University, Newcastle upon Tyne.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147

Delveinsight

DECEMBER 3, 2020

Janssen buys a gene therapy asset. Janssen buys a new gene therapy aimed at reversing a severe disease, from Hemera Biosciences. AMD Patients often have low levels of CD59, which is a protein that protects the retina from damage caused by a vital part of the body’s natural immune response called complement.

Gene Therapy 52

Gene Therapy Gene Antibody Research 52

Cloudbyz

MARCH 6, 2024

Unlike traditional small molecule drugs, which are chemically synthesized, biologics are produced through complex biotechnological processes, often involving recombinant DNA technology. This allows for the creation of highly specific and targeted therapies that can modulate biological pathways with remarkable precision.

Gene 99

Gene Immune Response Manufacturing Gene Therapy 99

XTalks

NOVEMBER 30, 2023

More Investigational Therapies for HIV Are on the Way American Gene Technologies, a pioneering biotech company headquartered in Rockville, Maryland, has released promising results from its Phase I trial of AGT103-T, a gene therapy designed for individuals with chronic HIV disease. In November 2023, Hookipa Pharma Inc.,

FDA Approval 111

FDA Approval Gene Therapy Gene Clinical Trials 111

pharmaphorum

OCTOBER 12, 2022

The payload, Del Bourgo tell us, is the therapeutic DNA or RNA sequence that will cure or fix damaged cells. Inactive viruses often act as the viral vector, as they’re able to deliver genetic therapies efficiently. We believe that genomic therapies are going to be important arms in our arsenal to treat many diseases.”.

Genome 105

Genome Genomics Medicine Development 105

The Pharma Data

DECEMBER 27, 2020

Taysha Gene Therapies announced that Queen’s University in Ontario, Canada, received Clinical Trial Application (CTA) approval from Health Canada for its Phase I/II trial of TSHA-101 for infantile GM2 gangliosidosis. RESTORE-1 is a Phase II trial of an intracerebral AAV-based gene therapy for Parkinson’s disease.

Trials 52

Trials Antibody Gene Therapy Gene 52

WCG Clinical

MAY 28, 2024

This proliferative capacity allows a rapid response to an incoming threat, such as a viral infection. T cells and B cells also express specialized antigen receptors that impart specificity to the immune response, directing immune attack against foreign “non-self” targets while sparing “self” targets expressed by healthy tissue.

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials FDA Approval 40

Roots Analysis

MAY 21, 2024

Several LNP-based therapies are currently being evaluated in the clinical trials; majority of these are conducted for treating breast cancer, ovarian cancer and lung cancer. In addition, these can be modified with ligands in order to improve the specificity and selectivity of the gene therapy and reduce off-target effects.

Drug Delivery 40

Drug Delivery Drugs Gene Therapy Bioavailability 40

The Pharma Data

DECEMBER 20, 2020

Pfizer and BioNTech announced additional data on neutralizing antibody and T-cell responses from their Phase I/II trial of their COVID-19 vaccine conducted in Germany. FLT180a is an AAV gene therapy. The product is an AAV9-based gene therapy for GM1 gangliosidosis. The vaccine only requires a single dose.

Trials 52

Trials Gene Therapy Vaccination Vaccine 52

pharmaphorum

JULY 26, 2022

At a basic level, the virus’s protein ‘coat’ and the enzymes it uses to gain control of the host cell’s DNA mutate frequently, making it difficult to target with therapeutics. Gene therapies are another approach that has been validated with approvals in recent years.

Gene Therapy 140

Gene Therapy Protein Vaccination Vaccine 140