Gene Therapy

FDA approves $4.25 million gene therapy for MLD

March 29, 2024 - Once again, the pharmaceutical industry has topped its existing astronomical prices with Lenmeldy, an FDA-approved gene therapy for children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD), which has a wholesale acquisition price tag of $4.25...

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Breaking Down the Cost of Sickle Cell Disease Gene Therapies

by Veronica Salib

Earlier this week, the Biden–Harris administration announced its plans to address the expensive price tag of sickle cell disease, looking for a way to widen access despite the cost. In an...

Exploring Gene and Cell Therapy Integration in Pharmacies

by Alivia Kaylor

After seeing promising results in cell and gene therapy clinical trials, cell and gene therapy is transforming patient care, offering new possibilities for treating diseases that previously had limited treatment options. As pharmacies...

A Pulse Check on Advancements in the Life Sciences, Pharma Industry

by Alivia Kaylor

In the life sciences and pharmaceutical industry, 2023 has seen remarkable advancements and breakthroughs that hold great promise for the future of healthcare. In a recent podcast episode of the Healthcare Strategies, Senior Editor Alivia...

FDA Launches New Advisory Committee for Genetic Metabolic Diseases

by Veronica Salib

Adding to the lengthy list of advisory committees under its belt, the United States Food and Drug Administration (FDA) announced its plans to create a new advisory committee to specialize in genetic...

FDA Approves First CRISPR-Based Gene Therapy

by Veronica Salib

Last week marked a monumental regulatory decision for CRISPR-based gene editing technologies. The United States Food and Drug Administration (FDA) approved the first two cell-based gene therapies for...

Evaluating the Breast Cancer Therapeutics Market

by Veronica Salib

As breast cancer awareness month continues, gathering a comprehensive understanding of the breast cancer therapeutics market is critical, including current trends and market players, gaps, and predictions for the coming year. Understanding...

FDA Clears IND for New Parkinson’s Disease Treatment Using IPSCs

by Veronica Salib

Today, Aspen Neuroscience announced that the United States Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for a new Parkinson’s Disease gene...

FDA Grants Bluebird Bio Priority Review of BLA for SCD Treatment

by Veronica Salib

On June 21, 2023, Bluebird Bio issued a press release announcing that the FDA had granted its biological license application (BLA) for a new sickle cell disease (SCD) treatment priority review...

FNIH Enhances Rare Disease Research by Adding 8 Diseases to BGTC

by Veronica Salib

On May 16, 2023, the Foundation for the National Institutes of Health (FNIH) announced eight rare diseases to be explored by the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC). As one of the newest additions...

Vertex and CRISPR Submit BLA for Sickle Cell Disease Treatment

by Veronica Salib

On April 3, 2023, Vertex Pharmaceuticals and CRISPR Therapeutics announced that they submitted Biological License Applications (BLAs) for an investigational treatment using exagamglogene autotemcel...

Pfizer Acquires Abzena’s Biologic Manufacturing Facility

by Hayden Schmidt

The contract development and manufacturing organization (CDMO) Abzena and Pfizer’s CDMO arm CenterOne reached an agreement last week, under which Pfizer will take control of Abzena’s...

From COVID to Cancer, Understanding the Applications of mRNA Vaccines

by Veronica Salib

Throughout the COVID-19 pandemic, patients, researchers, and providers became increasingly familiar with mRNA vaccines, which have unparalleled public health benefits. While mRNA vaccines have been researched for years, the rapid...

Scientists Explore HIV Treatments, Drugs, Vaccines, and Gene Therapy

by Veronica Salib

Human immunodeficiency virus (HIV), a viral infection that attacks and weakens the immune system, impacts 38.4 million people worldwide. According to the WHO, in 2021, 650,000 people globally died of an HIV-related illness. Despite high...

Eli Lilly Acquires Akouos in Approximately $487 Million Transaction

by Veronica Salib

In a recent press release from Eli Lilly and Company, the company announced that it had completed the acquisition of Akouos. Earlier this year, on October 18, 2022, Lilly announced plans to acquire the...

FDA Approves $3.5 Million per Dose Hemophilia B Gene Therapy

by Hayden Schmidt

Last week, the FDA’s Center for Biologics Evaluation and Research announced the approval of Hemgenix, an adeno-associated virus vector-based gene therapy for adults with hemophilia B (congenital...

FDA Approves Cell-Based Gene Therapy for Patients with Beta Thalassemia

by Veronica Salib

On August 17, 2022, the FDA approved bluebird bio's Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy to treat patients with beta thalassemia who need regular red blood...

Bluebird Bio’s Rare Disease Gene Therapies Recommended for FDA Approval

by Hayden Schmidt

Last week, the FDA Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 15 – 0, concluding that the benefits of bluebird bio’s gene therapy to treat cerebral...

UC Berkeley Loses CRISPR Gene-Editing Patent Appeal

by Samantha McGrail

The US Patent and Trademark Office (PTAB) recently ruled that CRISPR gene-editing technology belongs to the Broad Institute of Harvard and MIT, according to multiple news sources. After an...

The Center for Breakthrough Medicines, UPenn Boost Gene Therapies

by Samantha McGrail

The Center for Breakthrough Medicines (CBM) and the University of Pennsylvania Gene Therapy Program (GCP) recently collaborated to advance the discovery and manufacturing of gene therapies. The...

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