Cell & gene therapy

Five minutes of hands-on bench time now separates researchers from purified plasmid DNA using a new automated system from Thermo Fisher Scientific. The company’s cartridge-based KingFisher PlasmidPro automates the traditionally hours-long maxi-prep process. The KingFisher PlasmidPro benchtop instrument offers walk-away operation. Kevin Lowitz, vice president and general manager for sample prep at Thermo Fisher Scientific,…

Emory University stem-cell biologist Chunhui Xu is looking to space for a new way to mend damaged hearts on Earth. Her team is using the International Space Station (ISS) National Lab to test if the near-weightless environment of microgravity can make heart cells divide faster and survive longer, a novel approach borrowed from cancer researchers…

An MIT research group has unveiled a set of synthetic gene circuits designed to give scientists more  control over gene-therapy treatments, according to a recent paper. The study, appearing in the journal Cell Systems, describes genetic constructs meant to fine-tune how and when therapeutic genes are switched on inside a patient’s cells, aiming to keep…

A novel gene editing tool named STITCHR, developed at Mass General Brigham and collaborating institutions, overcomes central limitations of current technologies by enabling the scarless insertion of entire genes or large DNA fragments up to 12.7 kb. Based on reprogrammed retrotransposon activity guided by a Cas9 nickase and using RNA templates, STITCHR shows potential for…

Cell therapies hold immense promise but, traditionally, carry a similarly large price tag often linked to complex, manual manufacturing. In its latest move to help make these life-saving  and -enhancing treatments more scalable and potentially accessible, Cellares is partnering with the University of Wisconsin School of Medicine and Public Health to automate the clinical-scale manufacturing…

MIT engineers have developed an efficient method to directly convert skin cells into functional neurons, bypassing the time-consuming step of creating stem cells first. This streamlined process, achieving over 1000% yield in mouse cells, promises to accelerate the production of neurons for cell therapies. The research, which was published in Cell Systems, holds promise for…

One of the largest unmet needs in modern medicine is the effective treatment of osteoarthritis (OA)—a degenerative joint disease afflicting more than 30 million Americans and costing the healthcare system $459.5 billion in all-cause medical costs, according to a 2020 study in the Archives of Physical Medicine and Rehabilitation. “We see this as one of…

In the classic Bill Murray comedy Groundhog Day, the main character is stuck reliving the same day—again and again. Biotech startups often face a similar fate when they sprint to first-in-human trials without planning for the realities of large-scale manufacturing. “Many times, researchers rush to get their product tested in humans with processes that are…

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 cell and gene therapy (CGT) patent landscape reveals a perhaps surprising leader: the German biopharmaceutical company, Immatics Biotechnologies GmbH. As GlobalData has noted, the firm is one of the top filers of patents for CAR-T cell-based therapies. This contrasts with the broader oncology patent space, where well-known Swiss companies like Novartis and Roche have…

YTD 2024 FDA approvals: By the numbers 44 Novel Drug Approvals As of December 5, 2024, the FDA had approved a total of 44 novel drugs, spanning a diverse array of therapeutic areas and patient populations. These new treatments address both widespread public health concerns, such as cardiovascular disease and COPD, and more narrowly defined…

Beckman Coulter Life Sciences has announced a new automated clone screening platform for biologic drug development. The Cydem VT system integrates several established technologies — microbioreactor arrays, liquid handling, and analytical measurements — into a single platform capable of monitoring up to 96 parallel bioreactors. The system provides continuous monitoring of pH, dissolved oxygen, and…

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…

After establishing her lab at Cornell in 2019 and achieving notable success with publications in top journals, Liz Kellogg, Ph.D., associate member in the department of Structural Biology at St. Jude Children’s Research Hospital received a unique opportunity. St. Jude Children’s Research Hospital had embarked on a major expansion of its structural biology and genome…

Immunotherapies, including cellular immunotherapies, have dramatically impacted the treatment of cancer. Recent advances in the field, such as autologous (patient-derived) chimeric antigen receptor (CAR)-T cell therapies, have yielded remarkable results in relatively rare blood-based cancers. These results have driven an explosion in the research and development of different cellular products, including both autologous and allogeneic…

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…

In a move to bolster its presence in the pioneering biotech hub of South San Francisco, Astellas Pharma has unveiled a $90 million West Coast Innovation Center. The newly constructed 154,000-square-foot facility consolidates the company’s previously dispersed Bay Area operations, including research, technical operations, medical and development and commercial teams, under one roof. “I want…

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…

[Updated May 10, 2024] In the face of rising R&D costs and growing pricing pressures from payers, the pharma and biotech sectors continue to transform to adapt to an evolving landscape. While workforce reductions persist in 2024 for some companies, major players like AbbVie, AGC Biologics, Amgen, Novartis and Thermo Fisher Scientific are demonstrating confidence…

Despite biopharma’s 2023 layoffs and challenges, innovation won your clicks last year with over 130,000 of them on our Pharma 50 report alone. But while giants dominated, your clicks showed disruptive tech wasn’t far behind. The next-most popular article was a roundup of 100 trailblazing cell and gene therapy companies with more than 80,000 views.…

Houston boasts many world-class assets that have made it a formidable player on the global stage. From the world’s largest medical complex to mission control for the cosmos, few other cities can compete with its diverse strengths. Houston is also home to the prestigious Rice University, renowned for its leading science and engineering programs. Houston,…

Founded in 2011, Caribou Biosciences is a pioneer in the development of CRISPR genome editing technologies, a field honored with the Nobel Prize in Chemistry in 2020. Co-founded by Jennifer Doudna, Ph.D., one of the Nobel laureates, and CEO Rachel Haurwitz, and two other CRISPR pioneers, the company has raised over $800 million in funding,…