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Preclinical Study Advances CRISPR-Cas9 Gene Editing for Wiskott-Aldrich Syndrome Towards Clinical Use

Preclinical Study Advances CRISPR-Cas9 Gene Editing for Wiskott-Aldrich Syndrome Towards Clinical Use

A comprehensive preclinical study has been published detailing the in-depth characterization of stem cell potency and genotoxicity for clinical-scale ex vivo CRISPR-Cas9 gene editing. This research, using Wiskott-Aldrich Syndrome as a model, established an integrated strategy for editing human hematopoietic stem cells (HSCs) and assessing genotoxicity. The study's findings have been instrumental in securing Orphan Drug Designation from both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). This designation is a critical step in accelerating the development and review of therapies for rare diseases. The research focuses on the practical aspects of moving gene editing from laboratory settings to clinical application, emphasizing the need to demonstrate not only the effectiveness of the edit but also the potency, genotoxicity, and consistency of the process at a clinical scale. The project was driven by Asma Naseem, with significant contributions from collaborators including...

CRISPR-Combo System Accelerates Gene-Edited Crop Regeneration, Addressing Agricultural Bottleneck

CRISPR-Combo System Accelerates Gene-Edited Crop Regeneration, Addressing Agricultural Bottleneck

Researchers from Texas A&M AgriLife Research, the University of Maryland, and the U.S. Department of Agriculture have developed a new system called CRISPR-Combo to significantly speed up the regeneration of gene-edited crops. Published in Nature Communications, this system addresses a major challenge in agricultural biotechnology: the slow and often unreliable process of growing a complete plant from gene-edited cells. CRISPR-Combo works by simultaneously editing a target gene and activating the plant's own morphogenic genes, which are responsible for cell division and development into roots, shoots, and full plants. Unlike previous methods that involved inserting additional growth-promoting genes or relying on external hormones, this new approach uses the existing CRISPR tool to enhance the activity of genes already present in the plant's genome. This method has shown promising results in various crops, including potato, citrus, strawberry, and poplar, leading to faster regeneration times and higher effic...

Opus Genetics Advances Gene Therapies for Inherited Retinal Diseases, Secures FDA Alignment for Phase 3 Trial

Opus Genetics Advances Gene Therapies for Inherited Retinal Diseases, Secures FDA Alignment for Phase 3 Trial

Opus Genetics is making significant strides in developing gene therapies for inherited retinal diseases (IRDs), specifically OPGx-LCA5 for LCA5-associated IRD and OPGx-BEST1 for BEST1-associated IRD. According to Dr. George Magrath, CEO of Opus Genetics, the OPGx-LCA5 program, targeting an ultra-rare disease affecting approximately 200 U.S. children, has shown promising results. All six participants treated to date (three pediatric and three adult) demonstrated improvement across various measures, including full-field stimulus threshold (FST) testing, best-corrected visual acuity (BCVA), virtual-reality maze tests, and microperimetry. Retinal sensitivity improved by about 1.5 log units at both blue and red wavelengths. The company has also secured FDA alignment for an eight-participant Phase 3 registrational trial for OPGx-LCA5, utilizing a 6-month pretreatment period where each participant serves as their own control. The OPGx-BEST1 program aims to restore bestrophin-1 channel function in the retinal pigm...

Coya Therapeutics Reaches 100th Participant Enrollment in ALS Trial for COYA 302

Coya Therapeutics Reaches 100th Participant Enrollment in ALS Trial for COYA 302

Coya Therapeutics, Inc., a clinical-stage biotechnology company, has announced the enrollment of its 100th participant in the Phase 2/3 ALSTARS Trial (NCT07161999) for COYA 302. This investigational drug, a combination of proprietary low-dose IL-2 and CTLA-4 Ig, is being evaluated for the treatment of amyotrophic lateral sclerosis (ALS). The company anticipates completing the full enrollment of 120 participants in the coming weeks, with topline data expected in early Q2 2027. COYA 302 has received FDA Fast Track designation for ALS treatment. The ALSTARS Trial is a randomized, multi-center, double-blind, placebo-controlled study designed to assess the efficacy and safety of COYA 302. Participants are randomized to receive one of two dosing regimens or a placebo over a 24-week double-blind treatment phase, followed by a 24-week blinded active-treatment extension phase. The primary endpoint focuses on changes in disease severity as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised...

FDA Approves Moderna's mFLUSIVA, First mRNA Flu Vaccine for Adults 50 and Older

FDA Approves Moderna's mFLUSIVA, First mRNA Flu Vaccine for Adults 50 and Older

The U.S. Food and Drug Administration (FDA) has approved mFLUSIVA (mRNA-1010), Moderna's mRNA-based influenza vaccine, for adults aged 50 years and older. This marks the first mRNA flu vaccine to receive FDA approval. The approval for adults aged 50 through 64 years is traditional, while for those 65 and older, it was granted through an accelerated pathway, requiring further confirmatory evidence of clinical benefit. The pivotal FLUENT phase 3 trial, which involved over 40,000 adults aged 50 and above, demonstrated that mFLUSIVA had a 26.6% greater relative efficacy against confirmed influenza-like illness compared to a licensed standard-dose influenza vaccine. The mRNA technology instructs cells to train the immune system, and its manufacturing process does not require the propagation of vaccine viruses in eggs, potentially allowing for faster production closer to the flu season. Side effects reported for mFLUSIVA are similar to other vaccines, including mild reactions such as fatigue, muscle and joint ac...

Healthcare Systems Must Prioritize 'Care' Over Operational Goals, Bioethics Experts Argue

Healthcare Systems Must Prioritize 'Care' Over Operational Goals, Bioethics Experts Argue

Bioethics experts are emphasizing a critical distinction between 'healthcare' and 'care,' arguing that while healthcare is an organized enterprise with legitimate operational goals like safety, efficiency, and quality, these should not overshadow the fundamental practice of care. Healthcare systems encompass hospitals, clinics, professionals, technologies, policies, payment structures, documentation, and workflows. The Institute of Medicine's report, 'Crossing the Quality Chasm,' outlined that healthcare should be safe, effective, patient-centered, timely, efficient, and equitable. However, experts contend that achieving these operational metrics does not automatically equate to good care. They define care as the practice of recognizing a person's needs, taking responsibility to respond, acting competently, and remaining attentive to how that person responds. This distinction is crucial because a healthcare process can succeed on its own terms while making it more difficult for clinicians, patients, and fa...

Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma

Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma

Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma

Representatives Introduce Bipartisan Bill to Permanently Authorize Rare Pediatric Disease Priority Review Voucher Program

Representatives Introduce Bipartisan Bill to Permanently Authorize Rare Pediatric Disease Priority Review Voucher Program

U.S. Representatives Gus Bilirakis (R-FL), Nanette Barragán (D-CA), Kat Cammack (R-FL), Jake Auchincloss (D-MA), Tom Kean, Jr. (R-NJ), and Kevin Mullin (D-CA) have introduced bipartisan legislation to permanently authorize the U.S. Food and Drug Administration’s (FDA) Rare Pediatric Disease Priority Review Voucher (PRV) Program. This program, established in 2012, incentivizes the development of treatments for children suffering from rare and devastating diseases. The PRV program awards a transferable priority review voucher to companies that successfully develop and receive FDA approval for qualifying treatments. Since its inception, the program has facilitated the market entry of treatments for 47 rare pediatric diseases, a significant increase from the four FDA-approved treatments that existed before the program. The current authorization for the program is set to expire in 2029. The proposed permanent authorization aims to provide long-term certainty for researchers and investors, encouraging continued ...

FDA-Cleared Oral Appliances Emerge as Key Alternative for Obstructive Sleep Apnea Treatment

FDA-Cleared Oral Appliances Emerge as Key Alternative for Obstructive Sleep Apnea Treatment

For individuals diagnosed with obstructive sleep apnea (OSA) who cannot tolerate CPAP therapy, FDA-cleared oral appliances are becoming a significant alternative. These custom-fitted devices are worn during sleep and mechanically position the jaw or tongue to keep the airway open, differing from over-the-counter snoring guards. While often searched as 'FDA approved,' most oral appliances receive 'FDA clearance' through the 510(k) pathway, indicating substantial equivalence to existing devices rather than a full approval process for high-risk devices. This distinction is important for understanding their regulatory status. The American Academy of Sleep Medicine recognized oral appliances as an option for mild-to-moderate OSA and for CPAP-intolerant patients as early as 1995. Recent advancements include the 2023 FDA 510(k) clearance for Vivos Therapeutics' CARE oral appliances for severe OSA, expanding their traditional indication beyond mild-to-moderate cases. Examples of cleared devices include the Meridia...

Wellness Influencers Promote Berberine as "Nature's Ozempic" in the U.S., Despite Traditional Use

Wellness Influencers Promote Berberine as "Nature's Ozempic" in the U.S., Despite Traditional Use

Wellness Influencers Promote Berberine as "Nature's Ozempic" in the U.S., Despite Traditional Use

TwoStep Therapeutics Secures $62.5 Million Series A Funding and FDA Clearance for Cancer Drug

TwoStep Therapeutics Secures $62.5 Million Series A Funding and FDA Clearance for Cancer Drug

TwoStep Therapeutics Secures $62.5 Million Series A Funding and FDA Clearance for Cancer Drug

SSRIs and Ketamine Offer Distinct Approaches to PTSD Treatment, Differing in Mechanism and FDA Status

SSRIs and Ketamine Offer Distinct Approaches to PTSD Treatment, Differing in Mechanism and FDA Status

Selective Serotonin Reuptake Inhibitors (SSRIs) and ketamine therapy represent fundamentally different approaches to treating Post-Traumatic Stress Disorder (PTSD). SSRIs, such as sertraline and paroxetine, are FDA-approved and recommended as first-line treatments, working by gradually increasing serotonin levels in the brain over several weeks. In contrast, ketamine acts on the brain's glutamate system, promoting neuroplasticity and often showing symptom relief within about two weeks. Ketamine is currently used off-label for PTSD and is not recommended by major clinical guidelines due to lower-quality evidence, though it is considered when first-line treatments are insufficient. Clinical trials indicate SSRIs help approximately 58% of individuals respond, while real-world data for ketamine therapy report higher response and remission rates, though no head-to-head controlled trials have been conducted. SSRIs are less effective for combat-related PTSD, a particularly challenging form of the condition. Side ...