Tagged News
FDA Grants Rare Pediatric Disease Designation to Relief Therapeutics' EB Treatment RLF-TD011
• The FDA has granted Rare Pediatric Disease designation to Relief Therapeutics' RLF-TD011 for epidermolysis bullosa, a rare genetic skin disorder affecting approximately 500,000 people worldwide.
• RLF-TD011 is a hypotonic acid-oxidizing solution with hypochlorous acid that provides antimicrobial and anti-inflammatory properties while creating a wound microenvironment conducive to healing.
• The designation could potentially lead to a Priority Review Voucher upon approval, which has significant value as demonstrated by a recent EB treatment voucher that sold for $155 million.
Related Clinical Trials:
Northwestern University
Posted 2/6/2023
AnnJi Pharmaceutical Reports Promising Phase 1/2a Results for AJ201 in SBMA, a Rare Neuromuscular Disease
• AnnJi Pharmaceutical's AJ201 demonstrated positive results in a Phase 1/2a trial for Spinal and Bulbar Muscular Atrophy (SBMA), showing improvements in physical function and muscle biomarkers after 12 weeks of treatment.
• The investigational drug reduced mutant androgen receptor levels by more than 50% in over half of treated patients and activated the Nrf2 pathway, supporting its proposed mechanism of action.
• With no FDA-approved treatments currently available for SBMA, AnnJi plans to advance AJ201 to Phase 3 trials, potentially offering hope for approximately 1 in 40,000 males affected by this rare genetic disorder.
Related Clinical Trials:
AnnJi Pharmaceutical Co., Ltd.
Posted 2/28/2023
Rainbow Children's Hospital Completes Eighth Zolgensma Gene Therapy for Spinal Muscular Atrophy in India
• Rainbow Children's Hospital in Secunderabad, India has successfully administered its eighth Zolgensma gene therapy treatment for Spinal Muscular Atrophy, a rare genetic neuromuscular condition affecting approximately 1,000 children in India.
• The one-time gene therapy, which costs approximately Rs 14 crore (USD 1.7 million) per dose, works by replacing the defective SMN1 gene using adeno-associated viral vector technology to enable production of essential SMN protein.
• Following treatment, patients require continuous monitoring by a multidisciplinary team and weekly laboratory investigations to identify potential adverse events and enable early intervention.
Telomir Pharmaceuticals Secures $3 Million Investment to Advance Novel Age-Reversal Drug for Rare Diseases
• Telomir Pharmaceuticals has secured $3 million in premium equity financing from its largest shareholder to advance Telomir-1, a first-in-class age-reversal molecule, toward an IND submission for rare disease indications by year-end.
• The company's lead candidate, Telomir-1, targets five fundamental biological drivers of aging and has shown promising preclinical results across multiple conditions including Progeria, Wilson's disease, and Type 2 diabetes.
• Telomir is also developing Telomir-Ag2, a novel stabilized Silver(II) compound with broad-spectrum antimicrobial activity against drug-resistant pathogens, addressing a global market projected to exceed $30 billion.
Worldwide Clinical Trials Joins WCG's Avoca Quality Consortium to Enhance Clinical Trial Standards
• Worldwide Clinical Trials has joined WCG's Avoca Quality Consortium (AQC), a collaborative network of over 200 life sciences companies focused on elevating clinical trial quality.
• The strategic partnership aims to enhance quality, efficiency, and regulatory compliance through industry collaboration and best practices, ultimately delivering faster and more reliable outcomes for customers.
• This membership aligns with Worldwide's commitment to quality assurance across its services, which include bioanalytical laboratory services, Phase I-IV clinical trials, and post-approval studies.
AstraZeneca Showcases Groundbreaking Cancer Research at ASCO 2025 with Two Plenary Presentations
• AstraZeneca will present over 80 abstracts at ASCO 2025, including two plenary presentations featuring camizestrant for HR-positive breast cancer and IMFINZI for early gastric cancer, marking their seventh consecutive year with plenary data.
• The SERENA-6 trial is the first positive Phase III study for a next-generation oral SERD in first-line HR-positive breast cancer, pioneering the use of circulating tumor DNA to guide treatment decisions.
• DESTINY-Breast09 data shows ENHERTU plus pertuzumab is the first treatment in over a decade to demonstrate superiority over standard care in first-line HER2-positive metastatic breast cancer.
Related Clinical Trials:
AstraZeneca
Posted 11/19/2021
Daiichi Sankyo Co., Ltd.
Posted 9/1/2015
New Genetic Findings Challenge Traditional Understanding of Coats' Disease
• Research led by Dr. David Abramson reveals that Coats' disease, traditionally considered unilateral, shows bilateral involvement when examined with fluorescein angiography, challenging long-held clinical beliefs.
• Genetic testing identified a variation in the LTBP2 gene associated with Coats' disease, providing new insights into the condition's genetic underpinnings and supporting the concept that single genes can influence multiple diseases.
• Treatment approaches combining laser photocoagulation and anti-VEGF therapy showed promising results in advanced cases, though researchers speculate effectiveness may be linked to specific telomere biology disorders.
Bristol Myers Squibb to Close Gene Therapy Manufacturing Facility in Libertyville, Illinois
• Bristol Myers Squibb has announced the closure of its viral vector production facility in Libertyville, Illinois, with operations being consolidated to Devens, Massachusetts, as part of a $2 billion cost-saving initiative.
• The facility, which was acquired by Bristol Myers Squibb two years ago and previously used by Novartis to manufacture the gene therapy Zolgensma, will begin layoffs on July 1 according to state WARN reports.
• Company representatives acknowledged the difficult decision to cease vector manufacturing at the Libertyville site, which they noted "has operated at the highest level for BMS Cell Therapy."
PharmaEssentia's Ropeginterferon Shows Promising Results in Phase 3 Trial for Essential Thrombocythemia
• PharmaEssentia will present positive Phase 3 SURPASS-ET trial results showing ropeginterferon alfa-2b-njft achieved significantly higher durable clinical response rates compared to anagrelide (42.9% vs. 6.0%; p=0.0001) in essential thrombocythemia patients.
• The trial demonstrated not only effective blood count control but also a measurable reduction in JAK2 mutation allele burden over 12 months, addressing an underlying disease mechanism that current treatments fail to target.
• Ropeginterferon alfa-2b-njft (marketed as BESREMi® for polycythemia vera) could potentially offer a new second-line treatment option for essential thrombocythemia, a rare blood disorder characterized by excessive platelet production.
Related Clinical Trials:
PharmaEssentia
Posted 8/25/2020
CHOP Researchers Develop Breakthrough AAV Vectors for Brain Gene Therapy at Lower Doses
• Researchers at Children's Hospital of Philadelphia have engineered new adeno-associated viral (AAV) vectors that can target brain cells at significantly lower doses than current therapies, potentially improving safety and reducing costs.
• Two novel capsids were identified: AAV-Ep+ for Batten disease treatment and AAV-DB-3 for Huntington's disease, both showing robust efficacy in preclinical models and human neurons derived from stem cells.
• The breakthrough could transform treatment for neurodegenerative disorders by enabling one-time precision therapies that require lower doses while maintaining therapeutic efficacy, with potential applications for other inherited disorders.