DelveInsight

Nov 19, 2024

FDA Approves AADC Deficiency Gene Therapy by PTC Therapeutics PTC Therapeutics, Inc. has secured FDA accelerated approval for KEBILIDI (eladocagene exuparvovec-tneq), marking a historic milestone as the first-ever gene therapy directly administered to the brain in the United States. This innovative therapy addre...

Nov 12, 2024

FDA Approves Autolus's AUCATZYL for Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia Autolus Therapeutics has achieved a significant milestone with FDA approval for AUCATZYL (obecabtagene autoleucel), a next-generation CAR T-cell therapy for adults with relapsed or refractory B-cell acute lymphoblastic le...

Nov 08, 2024

Cervical cancer remains a leading cause of death among women worldwide. While increased awareness and early diagnosis have significantly reduced fatalities over the years, the risk is still substantial. A major factor in the development of cervical cancer is persistent infection with Human Papillomavirus (HPV), whi...

Nov 05, 2024

Prolong Pharmaceuticals Secures FDA Fast Track for PP-007 in Stroke Therapy Prolong Pharmaceuticals, LLC, a clinical-stage biopharmaceutical company, announced that its investigational therapy, PP-007 (PEGylated carboxyhemoglobin, bovine), has been granted Fast Track designation by the FDA for the treatment of a...

Nov 01, 2024

Imagine a disease that stealthily incapacitates the heart, liver, and skeletal muscles—slowly breaking them down due to the absence of a crucial protein. That's the story of Pompe disease, also known as glycogen storage disease type II (GSDII). Caused by a gene mutation that blocks the body’s ability to break down ...

Oct 29, 2024

FDA Grants Orphan Drug Status to MDL-101 for LAMA2-CMD Treatment Modalis Therapeutics Corporation has received Orphan Drug Designation (ODD) from the FDA for MDL-101, its promising treatment for congenital muscular dystrophy type 1A (LAMA2-CMD). This designation is a significant step, aimed at fostering the deve...

Oct 18, 2024

In the ever-evolving world of liver disease, the shift from Nonalcoholic Steatohepatitis (NASH) to Metabolic Associated Steatotic Hepatitis (MASH) marks a revolutionary leap in our understanding and approach to treatment. This transition isn't merely a rebranding; it signifies a profound recognition of the intricat...

Oct 16, 2024

Pompe disease is a rare genetic disorder that presents a spectrum of severity, with varying rates of progression and ages of onset. Symptoms can appear anywhere from infancy to late adulthood, with earlier onset generally associated with more rapid progression and increased severity. At all ages, the disease is mar...

Oct 15, 2024

Astellas & AviadoBio Sign Exclusive Deal for Gene Therapy AVB-101 in Frontotemporal Dementia AviadoBio Ltd. and Astellas Pharma Inc. have announced a strategic partnership under an exclusive option and license agreement for AVB-101, an investigational AAV-based gene therapy currently in Phase I/II developmen...

Oct 09, 2024

Nonalcoholic steatohepatitis (NASH), also referred to as Metabolic Dysfunction-Associated Steatohepatitis (MASH), has rapidly become a significant contributor to liver disease in the United States. The NASH market is growing at a substantial rate, driven by the increasing prevalence of the condition. According to t...