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Cellares and Seoul National University Hospital Partner for Automated Gene-Modified HSPC Therapy Manufacturing

Cellares and Seoul National University Hospital Partner for Automated Gene-Modified HSPC Therapy Manufacturing

Cellares and Seoul National University Hospital Partner for Automated Gene-Modified HSPC Therapy Manufacturing
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US FDA Launches INTERACT Program to Streamline Biologic Product Development

US FDA Launches INTERACT Program to Streamline Biologic Product Development

The U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) has introduced the INTERACT (INitial Targeted Engagement for Regulatory Advice on CBER ProducTs) program. This new initiative aims to facilitate early engagement between potential sponsors and FDA staff, providing advice on critical issues in the initial stages of biologic product development. The program's objective is to accelerate product development by addressing sponsors' questions, removing regulatory roadblocks, and establishing a clearer path for bringing products to patients. According to CBER director Peter Marks, INTERACT meetings will help streamline development by guiding sponsors on preclinical testing requirements, manufacturing aspects for first-in-human trials, initial clinical development strategies, and device or assay design considerations. The INTERACT program will replace the existing pre-pre-investigational new drug (IND) meeting program for all biologic products.
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Scancell Holdings Secures Up to $25 Million in Debt Financing for iSCIB1+ Phase 3 Development

Scancell Holdings Secures Up to $25 Million in Debt Financing for iSCIB1+ Phase 3 Development

Scancell Holdings Secures Up to $25 Million in Debt Financing for iSCIB1+ Phase 3 Development
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FDA Grants Breakthrough Therapy Designation to Glycomine's GLM101 for Rare Disease PMM2-CDG

FDA Grants Breakthrough Therapy Designation to Glycomine's GLM101 for Rare Disease PMM2-CDG

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to GLM101, an investigational drug by Glycomine, Inc., for the treatment of phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG). PMM2-CDG is a severe, multisystem rare disease with no approved treatments, affecting approximately 1 in 30,000 to 1 in 40,000 births in Europe and the United States. GLM101 is a mannose-1-phosphate replacement therapy designed to address the underlying deficiency in mannose-1-phosphate caused by genetic mutations in PMM2, which disrupts glycosylation. This designation is based on compelling clinical evidence from Glycomine's open-label Phase 2a study, which showed improvements in ataxia and other clinical measures after 24 weeks of GLM101 treatment. The drug is currently being evaluated in a global Phase 2b study, with topline data expected in the fourth quarter of 2026.
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Artificial Intelligence Transforms Gene Therapy Development, Addressing Key Challenges in U.S. Healthcare

Artificial Intelligence Transforms Gene Therapy Development, Addressing Key Challenges in U.S. Healthcare

Artificial Intelligence (AI) is revolutionizing the field of gene therapy, impacting every stage from target discovery to clinical translation. AI technologies, including machine learning, deep learning, and generative AI, are being applied to analyze vast amounts of biological data generated by high-throughput sequencing and multi-omics platforms. This allows for more efficient identification of disease-causing genes, optimization of genome-editing strategies like CRISPR-Cas systems, and the design of novel viral capsids and synthetic delivery systems. For instance, AI-guided directed evolution is significantly reducing the experimental search space for vectors with improved tissue-specificity and immune evasion capabilities. In manufacturing, AI models are being developed to monitor and control bioprocess parameters, aiming to improve the yield and consistency of complex biological products such as viral vectors. The U.S. Food and Drug Administration (FDA) is actively adapting its regulatory frameworks t...
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US FDA approves Lilly's once-weekly insulin injection for type 2 diabetes

US FDA approves Lilly's once-weekly insulin injection for type 2 diabetes

Sept 24 (Reuters) - The US Food and Drug Administration has approved Eli Lilly's once-weekly basal insulin, Onswik, for adults with type 2 diabetes, the drugmaker said on Thursday, offering patients an alternative to daily long-acting insulin injections.
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Former Johnson Johnson CEO Alex Gorsky Invests in AI Biotech Enveda's $311 Million Funding Round

Former Johnson Johnson CEO Alex Gorsky Invests in AI Biotech Enveda's $311 Million Funding Round

Former Johnson Johnson CEO Alex Gorsky Invests in AI Biotech Enveda's $311 Million Funding Round
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FDA Panel Endorses Grail's Multi-Cancer Blood Test for Individuals Aged 50 and Older

FDA Panel Endorses Grail's Multi-Cancer Blood Test for Individuals Aged 50 and Older

A federal advisory panel to the Food and Drug Administration (FDA) has endorsed Grail Inc.’s Galleri blood test, designed to detect signs of various cancers in apparently healthy individuals aged 50 and older. The panel voted 7-2, with one abstention, concluding that the DNA-based test offers more potential benefits than risks, despite acknowledging that more study is needed. The Galleri test does not diagnose cancer directly but identifies signals that may indicate the presence of one of over 50 types of tumors. A positive result necessitates further medical evaluation to confirm a cancer diagnosis. This endorsement marks the first time a federal advisory group has backed such an experimental multi-cancer blood test. The FDA is not bound by the panel's advice but typically considers it heavily in its final decisions, which are expected in a few months.
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Ionis and Roche Announce Positive Interim Phase 3 Data for Sefaxersen in IgA Nephropathy

Ionis and Roche Announce Positive Interim Phase 3 Data for Sefaxersen in IgA Nephropathy

Ionis and Roche Announce Positive Interim Phase 3 Data for Sefaxersen in IgA Nephropathy
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Moderna's mRNA Vaccine Shows Promise in Reducing Melanoma Recurrence and Treating Other Cancers

Moderna's mRNA Vaccine Shows Promise in Reducing Melanoma Recurrence and Treating Other Cancers

Moderna's mRNA vaccine, developed in partnership with Merck, has demonstrated significant success in reducing the recurrence of melanoma in large-scale trials. This development is part of a broader effort by Moderna to expand its focus beyond COVID-19 vaccines into oncology and rare diseases. The vaccine works by rapidly activating patients' immune systems to target cancer cells. Beyond melanoma, the company is also reporting promising results in treating other challenging cancers, including glioblastoma, pancreatic, lung, and breast cancers. This expansion into various cancer types highlights the versatility and potential of mRNA technology in therapeutic applications. The ongoing research and clinical trials aim to validate these findings and bring new treatment options to patients with these difficult-to-treat conditions.
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Viking Therapeutics' VK2735 Shows Promising Weight Loss Maintenance in Clinical Trials

Viking Therapeutics' VK2735 Shows Promising Weight Loss Maintenance in Clinical Trials

Viking Therapeutics announced positive topline results for its injectable GLP-1/GIP dual agonist, VK2735, demonstrating significant weight loss and impressive maintenance of that weight loss in a Phase 1 clinical trial. After 21 weeks, participants receiving 17.5 mg of VK2735 experienced an average body weight reduction of 17.7%. Crucially, the study also evaluated various maintenance dosing options. Following a transition to every-other-week dosing for three months, up to 97% of the weight loss was maintained, compared to 61% for the placebo group. Monthly dosing maintained up to 90% of the weight loss. An exploratory control arm that continued weekly dosing showed a 21.7% weight loss, with Viking suggesting further weight loss could occur with longer-term weekly administration. The safety profile was favorable, with gastrointestinal-related adverse events during the 12-week maintenance period being comparable to placebo, and no moderate or severe nausea reported in the step-down regimens.
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AI Becomes Essential in Biopharma for Accelerating Drug Development and R Pipelines

AI Becomes Essential in Biopharma for Accelerating Drug Development and R Pipelines

AI Becomes Essential in Biopharma for Accelerating Drug Development and R Pipelines