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Haemophilia drug could reduce treated bleeding episodes
Once-weekly or once-monthly dosing of Mim8 provides optionality and flexibility for people living with haemophilia A with or without inhibitors, stated Novo Nordisk’s EVP for Development.
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Modifier gene therapy – clinical development and manufacturing considerations
Expanding on his earlier podcast discussion with EPR, Dr Arun Upadhyay, Chief Scientific Officer and Head of Research & Development at Ocugen, discusses the company’s promising modifier gene therapy candidates for ophthalmic disorders.
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Analysis of chiral compounds using supercritical fluid chromatography
Sub/supercritical fluid chromatography is becoming a “dominant” technique for enantiomeric separation, especially purification, the researchers explained.
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Five-year data from Genentech SMA study released
Spinal muscular atrophy (SMA) patients in the long-term extension Evrysdi study maintained or improved key developmental skills over the five-year period, data shows.
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First-in-class telomerase inhibitor approved for blood cancer
The FDA’s regulatory approval of the telomerase inhibitor is welcome, considering the high unmet need for many lower-risk myelodysplastic syndromes (LR-MDS) patients, according to Geron.
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Combination therapy may hold potential for severe viral hepatitis
New findings from a Phase II study suggest the curative potential of a combination treatment for chronic hepatitis delta virus, according to Gilead Sciences.
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Advancing targeted treatments for multiple myeloma
Edmond Chan, Senior Director, EMEA Therapeutic Area Lead, Haemato-Oncology, Johnson & Johnson Innovative Medicines, offers insight into the promising evidence of cell therapies and biologic-based treatments for patients with multiple myeloma.
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Innovating small molecule injectables: market forecast to 2033
Key trends in the global small molecule injectable drugs market up to 2033 include advancements in technology, drug delivery systems and fast-track approvals of new drug formulations, research predicts.
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Biologic demonstrates durable tumour regression in rectal cancer
The updated, longer-term Phase II trial results suggest that the antibody therapy could offer a novel approach for treating advanced rectal cancer.
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Technology innovation aiding process spectroscopy market growth
Research suggests that the process spectroscopy market will gain a compound annual growth rate (CAGR) of 5.33 percent by 2033.
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AstraZeneca immunotherapy could boost lung cancer survival
Over half of patients were alive three years post-treatment with the monoclonal antibody (mAb) in AstraZeneca’s Phase III trial, data shows.
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Sustainable RP-HPLC method for simultaneous determination of hypertension drugs
The chromatographic method uses ethanol as a solvent and as such, provides an environmentally-friendly approach, the paper stated.
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Oligonucleotide ALS therapy approved in EU
The European Commission (EC) has granted Biogen its third rare disease treatment approval in the European Union.
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Pioneering AAV gene therapy for Krabbe disease
Dr Maria Escolar, Chief Medical Officer, Forge Biologics, explores the advantages of the company’s novel AAV gene therapy for Krabbe disease, which could help to overcome some of the immune and safety challenges the gene therapy sector is facing.
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LBP-immunotherapy could benefit oncology patients
New data for a microbiome-based therapeutic together with an immune checkpoint inhibitor has shown “encouraging clinical benefits” in advanced cancers.
