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Clinical Trial News

10x Genomics Partners with Singapore Institute to Launch AI-Driven Cancer Drug Discovery Study

AIDrug DiscoveryPrecision Medicine
  • 10x Genomics and A*STAR Genome Institute of Singapore announced the TISHUMAP project, an ambitious collaboration to analyze up to 2,500 clinical tissue samples using AI and spatial biology technology.
  • The study will focus on gastric, liver, and colorectal cancers, utilizing 10x Genomics' Xenium platform to visualize gene activity within individual cells in intact tissues.
  • The collaboration aims to accelerate drug target discovery and enable precision medicine by identifying novel biomarkers and therapeutic targets for cancer and inflammatory diseases.
  • Advanced AI will be combined with spatial omics data to detect critical patterns across large datasets, potentially leading to new diagnostics and personalized treatment plans.

BIRD Foundation Approves Additional Funding for iTOL-102 Type 1 Diabetes Cell Therapy Following FDA Pre-IND Meeting

  • The BIRD Foundation approved a fifth milestone payment of approximately $166,000 USD to support continued development of iTOL-102, a potential cure for Type 1 diabetes that would eliminate the need for lifelong immunosuppression.
  • The funding follows a successful Type B Pre-IND meeting with the FDA in February 2025, where the agency provided constructive guidance on the clinical development pathway for First-in-Human trials.
  • iTOL-102 combines Kadimastem's IsletRx stem cell-derived insulin-producing islet-like cells with iTolerance's iTOL-100 immunomodulatory platform, demonstrating functional insulin secretion and disease reversal in preclinical diabetic animal models.
  • The companies are currently preparing for safety toxicology studies and clinical trial submissions based on FDA guidance, with total BIRD Foundation support reaching nearly $882,352 USD to date.

Jaya Biosciences Secures Key Asian Patents for Novel Alzheimer's Gene Therapy Targeting Lysosomal Enzyme Defects

Precision Medicine
  • Jaya Biosciences received patent allowances in Japan, China, and Singapore for methods of detecting, preventing, reversing, and treating neurological diseases including Alzheimer's disease.
  • The company's lead therapy JB111 is an experimental CNS-directed AAV9-mediated gene therapy targeting PPT1 haploinsufficiency in Alzheimer's patients.
  • Research from Washington University shows that heterozygous loss-of-function mutations in lysosomal enzyme genes represent significant risk factors for adult-onset neurological diseases.
  • The patents cover major Asian markets where Alzheimer's disease affects over 22 million people combined across Japan, China, and Singapore.

Hansa Biopharma Secures $24.3M Funding as IDEFIRIX Sales Surge 76% in Q2 2025

  • Hansa Biopharma completed a directed cash share issue of approximately $24.3M and restructured its NovaQuest debt agreement, extending cash runway into Q2 2026.
  • IDEFIRIX product sales increased 76% year-over-year in Q2 2025 to 47.8 MSEK, with first-half sales representing approximately 80% of full-year 2024 revenue.
  • The company remains on track to report data from two Phase 3 trials in the second half of 2025, including the ConfIdeS kidney transplantation study and GOOD-IDES-02 anti-GBM trial.
  • A Biologics License Application (BLA) submission to the FDA is expected in the second half of 2025 following the ConfIdeS data readout.

Nanochon Receives Health Canada Approval for First-in-Human Trial of 3D Printed Knee Cartilage Implant

  • Nanochon has received Health Canada approval for a first-in-human clinical trial of its 3D printed Chondrograft implant to treat articular cartilage defects in the knee.
  • The prospective, 10-patient early feasibility study will evaluate the safety and performance of the minimally-invasive implant in patients aged 22-60 with failed conservative therapy.
  • The trial aims to assess cartilage-bone matrix regrowth, improved knee function and pain reduction, and delayed need for arthroplasty in patients with femoral condyle and trochlea lesions.
  • This represents the first step toward a larger North American clinical program for addressing the unmet need in thousands of patients with chondral defects.

Cosmo Pharmaceuticals Launches First-in-Class Acne Treatment Winlevi in Southeast Asia

  • Cosmo Pharmaceuticals has launched Winlevi (clascoterone) cream 1% in Singapore and Malaysia, marking the first topical acne treatment with a novel mechanism of action in over 40 years.
  • The drug features clascoterone as the first commercially available topical androgen receptor inhibitor that acts directly in sebaceous glands without systemic anti-androgen effects.
  • Two Phase III studies demonstrated superior efficacy compared to vehicle cream in reducing both inflammatory and non-inflammatory lesions in patients with facial acne vulgaris.
  • The partnership with Hyphens Pharma will expand distribution across ten Southeast Asian countries, addressing unmet medical needs in a region where acne affects millions of patients.

Sarepta Therapeutics Implements $400 Million Cost-Cutting Plan Amid Regulatory Challenges for Gene Therapy

Rare Disease
  • Sarepta Therapeutics announced a $400 million annual cost-savings initiative in July 2025, including a 36% workforce reduction of 500 employees to achieve financial sustainability through 2027.
  • The company's Q2 2025 Duchenne muscular dystrophy franchise generated $513 million in net product revenue, with ELEVIDYS gene therapy contributing $282 million despite facing FDA-mandated black box warning for acute liver risks.
  • Sarepta is strategically pivoting from gene therapy to siRNA platform development, discontinuing most LGMD programs while advancing trials for FSHD, myotonic dystrophy, and Huntington's disease with Phase 3 readouts expected in 2026.

IHC Testing Enables Rapid TP53 Mutation Detection in MDS and AML Patients

Precision Medicine
  • Researchers identified a 7% p53 immunohistochemistry threshold that best correlates with multi-hit TP53 mutations in myelodysplastic syndromes and acute myeloid leukemia patients.
  • The IHC testing provides results within 48-72 hours compared to 2+ weeks for next-generation sequencing, enabling faster treatment decisions for high-risk patients.
  • Higher IHC positivity correlated with worse overall survival outcomes, though false-negative results occurred in all TP53 sole nonsense or frameshift mutations.
  • Early TP53 mutation identification allows clinicians to avoid ineffective intensive chemotherapy and expedite stem cell transplant planning for patients with poor prognosis.

Collection of Blood and/or Bone Marrow for Investigation and Characterization of Normal and Abnormal Cell Growth

NCT01174615Recruiting
University of Massachusetts, Worcester
Posted 9/1/2009

AAV Gene Therapy Pipeline Expands with 180+ Companies Developing 250+ Treatments Despite Recent Setbacks

Drug Approval
  • Over 180 companies are actively developing more than 250 AAV-based gene therapies, representing a robust pipeline for treating genetic disorders including hemophilia, spinal muscular atrophy, and inherited retinal diseases.
  • Recent FDA approvals include Roctavian for severe hemophilia A and Elevidys for Duchenne muscular dystrophy, demonstrating the therapeutic potential of AAV vectors in addressing rare genetic conditions.
  • The field has experienced notable setbacks, including Pfizer's termination of its hemophilia A partnership and a patient death linked to Elevidys treatment, highlighting ongoing safety challenges.
  • Leading pipeline candidates include DTX401 for glycogen storage disease, SRP-9003 for limb-girdle muscular dystrophy, and several innovative therapies targeting retinal diseases and cardiovascular conditions.

FDA Implements Major Workforce Reduction and Policy Changes Amid Regulatory Overhaul

Rare Disease
  • Up to 3,500 FDA staffers received termination notices following a Supreme Court ruling that found the government's HHS overhaul to be lawful.
  • FDA Commissioner Marty Makary proposed lowering prescription drug user fees and offering speedier reviews to companies willing to reduce drug costs.
  • The agency released over 200 complete response letters for transparency, revealing rejection rationales for previously approved therapies including Eli Lilly's Alzheimer's drug Kisunla.
  • Two rare disease therapy rejections were issued to Ultragenyx for Sanfilippo syndrome type A and Capricor Therapeutics for DMD-associated cardiomyopathy.

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