gene therapy

Novartis has released positive topline results from the phase 3 STEER trial evaluating OAV101 IT (intrathecal onasemnogene abeparvovec) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2, aged two to under 18 years. The STEER trial is a pivotal, sham-controlled study designed to evaluate OAV101 IT against a procedure that mimics drug administration without…

As 2024 drew to a close, the cell and gene therapy sector has witnessed a string of recent approvals and some positive clinical trial data such as Mesoblast’s Ryoncil receiving FDA authorization for pediatric graft-versus-host disease in December, Adaptimmune’s Tecelra gaining approval for synovial sarcoma in August, and the landmark clearance of Casgevy (Exagamglogene Autotemcel)…

The U.S. Food and Drug Administration (FDA) recently released a draft guidance titled “Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products.” This 40-page document, while not legally binding, offers insights into best practices for common questions received by the Office of Therapeutic Products (OTP). While each question is answered in brief, the…

The field of gene therapy has seen unprecedented growth in recent years, with 20 approved indications in the U.S., a record number of approvals in 2023, and a rapid expansion of investigational trials across various diseases. Great strides have been made in potential treatments for inherited and acquired genetic diseases, including hemophilia B, sickle cell…

Swiss pharma giants Novartis and Roche consistently rank among the top pharma companies in terms of revenue. In 2024, the two companies are also leading in total international patent volume. Meanwhile, smaller firms like Incyte and Vertex Pharma are punching above their weight in market impact. For instance, Incyte, with a market cap of just…

Nine months into 2024, the search for effective neurodegenerative disease therapies continues, yielding a mix of promising advances and frustrating setbacks. After something of a lull in the 1990s and early 2000s, research into novel treatments for neurodegenerative diseases has surged back to life. One factor propelling the trend is the uptick in neurodegenerative disease…

Imagine going from near-total blindness to seeing the world in a whole new light – literally. That’s the reality for some participants in a recent gene therapy trial for LCA1, where vision improvements ranged from a 100-fold increase to a 10,000-fold jump. LCA1 (Leber Congenital Amaurosis 1) is a type of Leber Congenital Amaurosis that…

 The biotech industry continues to be in something of a “hangover period” where everyone realizes that there was “probably just a little bit too much enthusiasm” in circa 2020 and 2021, said Matthias Kleinz, DVM, Ph.D., executive vice president, translational sciences at UPMC Enterprises, the innovation and commercialization arm of University of Pittsburgh Medical…

In an exclusive interview with Drug Discovery & Development, Astellas Pharma CEO Naoki Okamura and Chief Scientific Officer Yoshitsugu (Yoshi) Shitaka shared details about its new $90 million, 154,000-square-foot biotech facility in South San Francisco. The new Astellas facility will serve as a central hub for Astellas’ Bay Area employees and be pivotal for the…

Cell and gene therapies are upending the treatment of a growing number of diseases by addressing the underlying causes of genetic disorders. Yet the high costs associated with these therapies, sometimes costing multiple millions of dollars for a single treatment, pose significant challenges for patients, payers and healthcare systems. To address this matter, a growing…

Cell and gene therapies often promise unparalleled treatment options for patients, but sometimes those benefits come at an extraordinary cost. The therapy class is responsible for the world’s most expensive drugs, including the recently FDA-approved gene therapy, Libmeldy, with a wholesale acquisition cost of $4.25 million. The prior record holder, Hemgenix, had a $3.5 million…

Genetic tools are growing more powerful by the day and hold immense medical promise. Kristofer Mussar, managing director of VectorBuilder GmbH who holds a Ph.D. in molecular genetics and epigenetics, noted that in the wake of the pandemic, genetic research requires increased awareness and ethical stewardship to responsibly tap into its power. While the current…

As the year draws to a close, it is clear that molecular science and diagnostics is the hottest funding area in the biotech industry. In an analysis of 50 of the best-funded biotechs of 2023 focused on human health, molecular and science and diagnostics startups collectively attracting roughly $945 million, dwarfing the figures in other…

Prix Galien names 2023 winners in pharma and biotech In the world of medical innovation, few accolades carry as much weight as the Prix Galien Awards, which highlight the advances in biotech, pharmaceuticals, and other domains. The 2023 winners include Bristol Myers Squibb’s Camzyos (mavacamten) as the best biotechnology product and Lilly’s Mounjaro (tirzepatide) and…

From her early days as a research assistant at UCLA, to her work as a postdoctoral fellow at Stanford, Deepcell co-founder and CEO Maddison Mahdokht Masaeli has actively engaged in the field of biomedical engineering. Now, Masaeli and her team at Deepcell, a company she co-founded in 2017, are introducing a new strategy to drug…

The gene therapy platform company Capsida Biotherapeutics (Thousand Oaks, California) is gearing up to unveil initial data on its engineered adeno-associated virus (AAV) capsids at the upcoming American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, held May 16–20 in Los Angeles. In developing these capsids, Capsida Chief Scientific Officer Susan Catalano explained…

Cell and gene therapies are the fastest growing area for drug development. The groundwork for this category was laid with the mapping of the human genome in 2003, and the field has developed rapidly in the intervening decades. These powerful therapies have significant potential for the treatment of cancer and other previously “undruggable” diseases. But…

A gene therapy for spinal muscular atrophy (SMA) patients is making waves. Novartis (NYSE:NVS) revealed in a press release new long-term data highlighting the durability of Zolgensma (onasemnogene abeparvovec) up to 7.5 years after a single treatment. The data comes from two long-term follow-up studies, LT-001 and LT-002, which examined a range of patient populations…

In recent years, the cell and gene therapy market has grown at a rapid clip. The momentum is likely to continue. According to Vision Research Reports, the global cell and gene therapy market size could hit around $42.56 billion by 2030, growing at a CAGR of 39% from 2022 to 2030. The burgeoning demand in…

Biotechnology traces its roots to the Neolithic era when early civilizations discovered how to ferment grains to prepare alcoholic brews and bread. It wasn’t until genetic engineering became mainstream in the mid-1970s, however, that biotechnology, as we know it today, was launched. The field continues to evolve today. The advent of new technology has revolutionized the market…