New Stem Cell Method Could Advance Research Into Spinal Muscular Atrophy by Madaline Spencer| Published on: Oct 15, 2026 (+++)

New Stem Cell Method Could Advance Research Into Spinal Muscular Atrophy by Madaline Spencer| Published on: Oct 15, 2026 Kyle Loh, PhD, Associate Professor at Stanford Medicine, discusses research that could help scientists better understand spinal muscular atrophy (SMA) by generating new motor neurons from human hindbrain cells in the laboratory. https://checkrare.com/new-stem-cell-method-could-advance-research-into-spinal-muscular-atrophy/ Ultragenyx Sells PRV for $210M | The CheckRare Brief Ep 11 by CheckRare Staff| Published on: Oct 14, 2026 In this episode, we discuss Ultragenyx’s Priority Review Voucher that sold for $210 million, the efficacy of newborn screening on MLD, and ethical considerations needed to design and conduct clinical trials. https://checkrare.com/ultragenyx-sells-prv-for-210m-the-checkrare-brief-ep-11/ FDA Approves Gazyva for Idiopathic Nephrotic Syndrome, Offering a New Treatment Option by Madaline Spencer| Published on: Oct 14, 2026 Keisha L. Gibson, MD, MPH, pediatric nephrologist at the UNC School of Medicine, discusses the approval of Gazyva (obinutuzumab) for patients with childhood-onset idiopathic nephrotic syndrome (INS). https://checkrare.com/fda-approves-gazyva-for-idiopathic-nephrotic-syndrome-offering-a-new-treatment-option/

Breaking barriers in brain gene therapy Researchers are developing new delivery technologies to bring gene therapies to the central nervous system safely and precisely. Written byAlejandra Manjarrez, PhD

Breaking barriers in brain gene therapy Researchers are developing new delivery technologies to bring gene therapies to the central nervous system safely and precisely. Written byAlejandra Manjarrez, PhD https://www.drugdiscoverynews.com/breaking-barriers-in-brain-gene-therapy-17537?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-8NXoSueeDn6O6K_9XiFDwPNnhdy5h5vHqR1gfCQr_c0cAAMIZXcNhlIZ30u_45mxTL_Unra24blayXGq5HSNhc2FcOOw&_hsmi=444434988&utm_content=444434988&utm_source=hs_email By correcting, replacing, or silencing disease-causing genes, gene therapies offer the potential to treat disorders at their source rather than simply managing symptoms. Several gene therapies have already reached the clinic for inherited diseases, demonstrating the promise of genetic medicines. Yet, despite a few landmark approvals for central nervous system (CNS) disorders, widespread and precise delivery to the brain remains one of the field’s greatest challenges. The problem is often not the therapeutic gene itself, but how to deliver it. The brain is protected by multiple biological barriers that prevent most circulating molecules from entering brain tissue and reaching the intended targets. For Jingjing Gao, a biomedical engineer at the University of Massachusetts Amherst, tackling this challenge became a natural extension of her scientific journey. Trained as a chemical engineer, Gao’s doctoral research focused on designing novel materials capable of controlled drug release.

Emerging Therapeutic Modalities in Autoimmune Disease

https://bpsbioscience.com/emerging-therapeutic-modalities-in-autoimmune-disease?_hsenc=p2ANqtz-8Pe1R0YVjniJ6nwcnnOTIE4cSsHLkEzbuTFinxwBWqNQLBVE3w2ZaV5G0VBg446_FYXWA4z80m3iKZI02YtL5eGJ9B2g&_hsmi=444434988&utm_campaign=DDN_Newsletter_Dose&utm_content=444434988&utm_medium=email&utm_source=hs_email Autoimmune diseases are conditions characterized by dysregulated inflammation against self-antigens that should otherwise not trigger an immune response. Conventional therapy for autoimmune diseases suppresses general immune function to modulate this uncontrolled inflammation. The drawback is that these treatments are largely non-selective and thus blunt the overall immune response, placing patients at a higher risk for life-threatening infection. To overcome the limitation of conventional therapies, discovering new selective treatments that cause minimal disruption to immune homeostasis remains critical.

Experimental gene therapy could one day treat hydrocephalus The first proof-of-concept study showed that the gene therapy reduced the production of cerebrospinal fluid in the brains of mice. Written byAllison Whitten, PhD

Experimental gene therapy could one day treat hydrocephalus The first proof-of-concept study showed that the gene therapy reduced the production of cerebrospinal fluid in the brains of mice. Written byAllison Whitten, PhD https://www.drugdiscoverynews.com/experimental-gene-therapy-could-one-day-treat-hydrocephalus-17589?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-8ohNvHT9U8QmyV8VQdS_g85lHmXQ8RgxODkA8m4SEQo3ckMnrzOYOLhnbTvkngP3LV8Pyd6BJUEOZVpin3T5uh5qfheg&_hsmi=444434988&utm_content=444434988&utm_source=hs_email When too much cerebrospinal fluid (CSF) seeps into the cavities of the brain, the result is life-threatening hydrocephalus. As the fluid builds up, it puts pressure around the surrounding fragile brain tissue. The primary treatment option involves an invasive surgery that inserts a shunt to drain the fluid elsewhere in the body, though these can often fail down the line. New research from Yu Luo’s lab at the University of Cincinnati showed a new way forward that gets directly at the source. By delivering a new AAV gene therapy into CSF that targets the choroid plexus — the network of vascular tissue that produces CSF — the researchers reduced CSF in a mouse model of hydrocephalus.

How scientists used AI to create ultra-stable, freezer-free mRNA vaccines A novel AI-guided approach enabled accelerated discovery of thermostable mRNA vaccines, eliminating ultra-cold storage barriers to expand global vaccine access. Written byPriyom Bose, PhD

How scientists used AI to create ultra-stable, freezer-free mRNA vaccines A novel AI-guided approach enabled accelerated discovery of thermostable mRNA vaccines, eliminating ultra-cold storage barriers to expand global vaccine access. Written byPriyom Bose, PhD https://www.drugdiscoverynews.com/how-scientists-used-ai-to-create-ultra-stable-freezer-free-mrna-vaccines-17585 In mRNA vaccines, lipid nanoparticles (LNPs) act as delivery vehicles that protect fragile mRNA so that it can safely reach target cells and trigger an immune response. While these carriers are safe and scalable, their instability in liquid form limits shelf life and requires ultra-cold storage. To improve stability, researchers have traditionally added excipients such as sugars, salts, or polymers to LNPs. Shifting to solid-state, water-free formulations extend thermostability, but previous efforts were limited by a narrow range of formulation options and slow screening processes. Now, in a new study published in Nature Biotechnology, MIT engineers applied an AI-driven optimization framework to stabilize FDA-approved LNP formulations, specifically for the Moderna (SM-102-based) and Pfizer-BioNTech (ALC-0315-based) mRNA vaccines. These formulations helped retain 100 percent bioactivity at 37 degrees Celsius for over two months — eliminating the need for ultra-cold storage completely.

A new design framework for mRNA vaccines could improve efficacy and safety New findings suggest that the location of mRNA expression in the body plays a critical role in shaping T cell responses, opening the door to more precise and effective mRNA-based treatments. Written byBree Foster, PhD

A new design framework for mRNA vaccines could improve efficacy and safety New findings suggest that the location of mRNA expression in the body plays a critical role in shaping T cell responses, opening the door to more precise and effective mRNA-based treatments. Written byBree Foster, PhD https://www.drugdiscoverynews.com/a-new-design-framework-for-mrna-vaccines-could-improve-efficacy-and-safety-17156?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-8BkiQlfgKupbgtgkPrIsp4q-9MnAX2SQCInw2rIx_ZYNLoWoNASkJ_EtXseBqAy-9zexcz-OjEDUIc1telNsOwcIVzqg&_hsmi=444112830&utm_content=444112830&utm_source=hs_email A new study from the Icahn School of Medicine at Mount Sinai challenges a central assumption in mRNA vaccine biology, showing that immune cells are not the only — or even the primary — determinant of vaccine effectiveness. Instead, non-immune cells such as muscle and liver cells play a previously underappreciated role in shaping immune responses, with major implications for mRNA vaccines and therapeutics. The preclinical research, published in Nature Biotechnology, also introduces a programmable system that allows scientists to control which cell types express mRNA-encoded proteins. In mouse models, this approach significantly improved the efficacy of an mRNA cancer vaccine for lymphoma.

Personalized vaccines work, making them is the hard part The science behind personalized mRNA cancer vaccines is working. Building a different drug for every patient, at scale, is the real fight. Written byAndrea Corona

Personalized vaccines work, making them is the hard part The science behind personalized mRNA cancer vaccines is working. Building a different drug for every patient, at scale, is the real fight. Written byAndrea Corona https://www.drugdiscoverynews.com/personalized-vaccines-work-making-them-is-the-hard-part-17504?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-9eS0EPQFrjU-Yg2KhEbctQkSQC34VQbtMFdNgjjeQN7m5gfruBGIAJ6bAKBges0B_7ijU1GWlzeb5wSfJSHu4gKTP0dA&_hsmi=444112830&utm_content=444112830&utm_source=hs_email Personalized mRNA cancer vaccines just cleared their biggest scientific hurdle. Recent Phase 3 data in melanoma showed the approach works in the clinic. But the possible implications of the findings are only as promising as the industry's ability to materialize them. "The payload works," Jeff Coller, RNA biologist at Johns Hopkins University, told DDN. "The carrier is the constraint." So here's the harder question nobody's fully answered yet, how do you actually make the thing? Every dose is a different molecule for a different person, so the industry can't just scale up the manufacturing playbook it built for COVID-19 vaccines. It has to build a new one.