Groundbreaking Intravenous Gene Therapy Offers New Hope for Metachromatic Leukodystrophy Patients
A recent study conducted by researchers from the TIDU GENOV at the Institut du Cerveau in Paris, France, presents promising results for the treatment of Metachromatic Leukodystrophy (MLD), a severe neurodegenerative disease. Led by Emilie Audouard with key...
Astellas’ Friedreich’s ataxia gene therapy cleared for clinical study after earlier version stumbled
After hitting a speed bump in its first attempt at developing a gene therapy for Friedreich’s ataxia, Astellas is ready to put a new version in the clinic. On Thursday, the Japanese drugmaker announced the FDA has cleared its IND for ASP2016. The company is hoping to...
Autolomous and BioCentriq Partner to Speed Up Cell Therapy Delivery
Collaboration leverages cutting-edge digital platform to streamline development and manufacturing activities and speed patient access to life-changing treatmentsLONDON and NEWARK, N.J., April 24, 2024 /PRNewswire/ -- Autolomous, the market-leading developer of...
Study reviews the role of a ribonucleic acid ‘MALAT1’ in hematological malignancies
Recent studies have shown that an abnormally high expression of MALAT1 in blood cancer cells could aid the chemotherapeutic resistance development through multiple intermolecular interactions. Although researchers first identified the role of MALAT1 in patients with...
FDA’s biologics chief Peter Marks previews accelerated approval guidance
The FDA’s Center for Biologics Evaluation and Research is planning to release some crucial guidance soon for CRISPR and gene therapy developers on platform technologies, accelerated approvals for rare diseases, and potentially in the “not-too-distant future,” a...
ImmunityBio’s ANKTIVA® Granted FDA Approval: Breakthrough IL-15 Receptor Agonist First-in-Class for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
The U.S Food and Drug Administration (FDA) has granted approval to ImmunityBio, Inc. (NASDAQ: IBRX) for their immunotherapy treatment, ANKTIVA® (N-803), in combination with Bacillus Calmette-Guérin (BCG), for patients with BCG-unresponsive non-muscle invasive bladder...
Ipsen and Skyhawk Therapeutics Announce Collaboration on RNA-Modulating Small Molecules for Rare Neurological Diseases
PARIS and BOSTON, April 22, 2024 – In a significant development in the biopharmaceutical industry, Ipsen and Skyhawk Therapeutics have entered into an exclusive global agreement to advance the discovery and development of novel small molecules that target RNA. These...
Duchenne UK and Parent Project Muscular Dystrophy Award $500,000 to Evaluate Safety and Tolerability of Muscle Progenitor Cells in Phase 1 Trial
WASHINGTON, April 23, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization, and the UK charity Duchenne UK, two leading organizations dedicated to ending Duchenne muscular dystrophy (Duchenne), are excited to announce the...
Bristol Myers, Cellares sign global CAR-T deal as biopharma companies look to diversify manufacturing partners
Cellares has inked its first international deal with Bristol Myers Squibb, with the cell therapy manufacturer noting it is going through “tremendous tailwinds” from the Biosecure Act with increased interest from many potential clients. Bristol Myers has reserved...
Lexeo Enters License Agreement for Friedreich Ataxia Treatment
LEXEO Therapeutics, Inc., a clinical stage genetic medicine company, entered an in-license agreement with Cornell University to expedite development of the investigational gene therapy candidate LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy. Under...
Capsida Biotherapeutics to Present New Data on its Wholly Owned Gene Therapy Programs in Genetic Epilepsy and Parkinson’s Disease at the Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
Company unveils Parkinson's disease associated with GBA mutations (PD-GBA) program; two oral presentations feature new data from Capsida's wholly owned programs in PD-GBA and genetic epilepsy due to STXBP1 mutations; both programs in IND-enabling studies to support...
5 Cell and Gene Therapy Decisions to Watch in 2024
In 2023, cell and gene therapy saw an unprecedented surge with seven FDA approvals, and this year, an even greater number of these treatments could reach the market. So far in 2024, the regulator has given the green light to three new CGTs, and at least seven...
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