XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing 58

Gene Editing Gene In-Vivo Clinical Trials 58

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

Delveinsight

FEBRUARY 11, 2021

However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinical trials. Avrobio gene therapy eradicates toxic substrate in Fabry patient. Enhancing the response to immuno-oncology in pancreatic cancer. They reported their observations in the journal JCI Insight.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

Delveinsight

JANUARY 12, 2021

Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor cells. The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52