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. The Orphan Drug Act creates incentives for developing orphan drugs to treat such diseases.

Gene Therapy 52

Gene Therapy Development Gene Drugs 52

XTalks

SEPTEMBER 27, 2023

Poseida is a clinical-stage biopharmaceutical firm that utilizes its unique non-viral gene engineering methods to develop innovative cell and gene therapies. She brings a wealth of knowledge from the biopharmaceutical realm, with expertise in oncology and allogeneic T-cell immunotherapy.

Life Science 59

Life Science Genetic Engineering Engineer Gene Therapy 59

NY Times

FEBRUARY 22, 2021

Two patients in a gene therapy study developed cancer years after treatment. It is not clear whether the therapy was responsible.

Gene Therapy 54

Gene Therapy Trials Research Gene 54

Roots Analysis

MARCH 16, 2023

In the last few years, researchers have become interested in using in vitro transcribed (IVT) mRNA as a drug delivery agent. The IVT mRNAs are structurally similar to natural mRNAs, these synthetic mRNAs can be used to express proteins through genetic engineering.

Vaccine 52

Vaccine Vaccination DNA Protein 52

The Pharma Data

JANUARY 10, 2021

FBX-101 is a first-in-human AAV gene therapy. Krabbe disease is a rare and fatal pediatric leukodystrophy caused by mutations in the galactosylceramidase (GALC) gene. The drug is an RNA interference (RNAi) therapeutic. The company also received an ok to launch a Phase I trial of TH1902, its lead peptide-drug conjugate.

Gene Therapy 52

Gene Therapy Trials Gene Clinical Trials 52

Roots Analysis

AUGUST 31, 2023

The applications of gene switch platforms can be broadly classified into the following two categories: Endogenous Gene Regulation Exogenous Gene Regulation Endogenous Gene Regulation The ability to regulate endogenous genes has various applications in biological research and gene therapy development.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

Advarra

JANUARY 11, 2024

Personalized medicine using genetic resequencing techniques, such as cell and gene therapies, enables researchers to create more customized therapies for combatting cancer. Adoptive T Cell Therapies Adoptive T cell therapy is another form of cancer treatment leveraging the patient’s immune system.

Vaccine 52

Vaccine Vaccination Protein Antibody 52

WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. WCG has many ways to support cell and gene therapy clinical trials for lupus.

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials FDA Approval 40

Advarra

OCTOBER 10, 2024

Another method of fostering antibiotic resistance is the prolonged low-level exposure to antibiotics, as is currently the case when people take prescribed antibiotics and excrete the unmetabolized drugs into the sewer. Also consider: Half the antibiotics produced in the U.S.

Bacteria 52

Bacteria Genetics Genetic Engineering Gene 52

NY Times

SEPTEMBER 14, 2021

Such treatments are extraordinarily promising and costly. Will the Biden administration commit to spending that could speed clinical trial results?

Gene Therapy 93

Gene Therapy Clinical Trials Clinical Trials Gene 93

XTalks

NOVEMBER 29, 2024

Their pipeline includes P-BCMA-ALLO1, a promising therapy targeting multiple myeloma that has received Regenerative Medicine Advanced Therapy designation and Orphan Drug designation from the US Food and Drug Administration (FDA). Meanwhile, Novartis acquired Kate Therapeutics in a $1.1

Gene Therapy 100

Gene Therapy Gene Gene Editing Genetic Engineering 100