MedPath

Tagged News

VeonGen Therapeutics Receives FDA Rare Pediatric Disease Designation for Stargardt Disease Gene Therapy VG801

Rare Disease
  • VeonGen Therapeutics (formerly ViGeneron) announced FDA Rare Pediatric Disease Designation for VG801, a dual AAV gene therapy targeting Stargardt disease and ABCA4-related retinal disorders.
  • The company is advancing two first-in-class gene therapies in clinical trials, with VG801 currently in a Phase 1/2 trial and functional endpoint development through FDA's RDEA program.
  • VeonGen's proprietary platforms include vgRNA REVeRT for delivering large genes exceeding AAV cargo limits and vgAAV for enhanced capsid delivery in ophthalmology applications.
  • Stargardt disease affects approximately 1 in 8,000 to 10,000 people worldwide and represents the most common inherited retinal disease with no approved treatments.

Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa

NCT06591793RecruitingPhase 1
AAVantgarde Bio Srl
Posted 7/2/2024

A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease

NCT05956626RecruitingNot Applicable
Ocugen
Posted 8/25/2023

An Observational Study in Children and Adults With Stargardt Disease

NCT06591806Recruiting
AAVantgarde Bio Srl
Posted 5/1/2024

A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

NCT07161544Not Yet RecruitingNot Applicable
AAVantgarde Bio Srl
Posted 9/1/2025

Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)

NCT07002398RecruitingPhase 1
ViGeneron GmbH
Posted 12/23/2024

Scinai Immunotherapeutics Surges 104% Following Italian Regulatory Clearance for Pincell Acquisition

Monoclonal AntibodyOrphan DrugRare Disease
  • Scinai Immunotherapeutics shares soared 104% to $5.15 after receiving Italian government clearance under Golden Power regulation to potentially acquire 100% of Italian biotech Pincell S.r.l.
  • The acquisition would add PC111, a monoclonal antibody targeting the Fas/FasL pathway for severe skin conditions including Pemphigus, Stevens-Johnson Syndrome, and Toxic Epidermal Necrolysis, which has received Orphan Drug Designation from the European Medicines Agency.
  • While the regulatory approval removes a major hurdle, the deal remains contingent on additional conditions including a €12 million grant decision expected by mid-July to early August 2025.
  • The move represents a significant expansion opportunity for Scinai, which operates dual business units in inflammation/immunology drug development and contract manufacturing services.

Mediar Therapeutics Initiates Phase 2 Trial of First-in-Class Anti-WISP1 Antibody for Idiopathic Pulmonary Fibrosis

Monoclonal AntibodyRare Disease
  • Mediar Therapeutics has dosed the first patient in its Phase 2 WISPer trial evaluating MTX-463, a first-in-class anti-WISP1 antibody for idiopathic pulmonary fibrosis treatment.
  • The randomized, double-blind, placebo-controlled study will measure changes in forced vital capacity at 24 weeks as its primary endpoint in IPF patients.
  • MTX-463 targets the myofibroblast, the key pathogenic cell driving fibrosis progression, representing a novel therapeutic approach for this devastating disease.
  • The company has entered a global licensing agreement with Eli Lilly to advance MTX-463 through the Phase 2 trial, while independently developing two additional fibrosis programs.

WISPer: Evaluation of MTX-463 in Participants With Idiopathic Pulmonary Fibrosis (IPF)

NCT06967805RecruitingPhase 2
Mediar Therapeutics
Posted 5/5/2025

Servier India Launches First-in-Class IDH1-Targeted Therapy Ivosidenib for Rare Blood and Bile Duct Cancers

Targeted TherapyDrug ApprovalOncologyRare DiseasePrecision Medicine
  • Servier India received CDSCO approval on May 14, 2025, to launch Ivosidenib (Tibsovo®), the first oral targeted therapy for IDH1-mutated acute myeloid leukemia and cholangiocarcinoma in India.
  • The therapy addresses a critical unmet medical need, as only 30-40% of AML patients in India receive adequate treatment and IDH1 mutations occur in approximately 7-14% of Indian AML patients.
  • Ivosidenib has already gained regulatory approval in over 42 countries, including FDA approval in 2019 and EMA approval in 2023 for specific patient populations.
  • The company is prioritizing accessibility and affordability through collaborations with healthcare providers, patient advocacy groups, and policymakers to ensure broader patient access across India.

FDA Denies Approval for Elamipretide, Forcing Stealth BioTherapeutics to Halt Production of Rare Disease Treatment

Drug ApprovalRare DiseaseOrphan Drug
  • The FDA has denied approval for Elamipretide, a treatment for Barth Syndrome that significantly improved quality of life for patients including a 17-year-old Mississippi teen who gained the ability to walk stairs and participate in normal activities.
  • Stealth BioTherapeutics will close operations after investing $100 million in the drug's development, with investors withdrawing support following the FDA's rejection.
  • Patients with Barth Syndrome, including Christopher Pena and his cousin Jacob Wilson, will lose access to the medication by the end of August, potentially leading to health deterioration.
  • Families are advocating for intervention from new FDA Commissioner Marty Makary and other officials, arguing the denial is due to regulatory red tape rather than safety or efficacy concerns.

FDA Awards Sarepta First Platform Technology Designation for Gene Therapy Vector

Rare Disease
  • The FDA has granted Sarepta Therapeutics a platform technology designation for its rAAVrh74 viral vector, marking one of the first such awards since the program launched in 2024.
  • The designation enables Sarepta to leverage existing safety and efficacy data from its approved Duchenne muscular dystrophy therapy Elevidys in future gene therapy applications using the same vector.
  • The platform technology program aims to streamline development and regulatory review processes for rare disease gene therapies by allowing companies to build upon previously established platform data.
  • Sarepta's rAAVrh74 vector is incorporated in multiple programs including the investigational limb-girdle muscular dystrophy treatment SRP-9003, which could be submitted for regulatory approval later this year.

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

NCT06246513Active, Not RecruitingPhase 3
Sarepta Therapeutics, Inc.
Posted 1/15/2024

A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT05126758Active, Not RecruitingPhase 3
Capricor Inc.
Posted 6/22/2022

Open-label Extension of the HOPE-2 Trial

NCT04428476Active, Not RecruitingPhase 2
Capricor Inc.
Posted 8/5/2020

A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)

NCT01976091CompletedPhase 1
Sarepta Therapeutics, Inc.
Posted 2/1/2015

LEGEND Study: EG-70 in NMIBC Patients BCG-Unresponsive and High-Risk NMIBC Incompletely Treated With BCG or BCG-Naïve

NCT04752722RecruitingPhase 1
enGene, Inc.
Posted 4/22/2021

A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

NCT05885412RecruitingPhase 1
Rocket Pharmaceuticals Inc.
Posted 8/29/2023

Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

NCT03652259TerminatedPhase 1
Sarepta Therapeutics, Inc.
Posted 10/27/2018

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT03406780CompletedPhase 2
Capricor Inc.
Posted 4/4/2018

Aspaveli Demonstrates Sustained 68% Proteinuria Reduction in Phase 3 VALIANT Trial for Rare Kidney Diseases

Rare Disease
  • Aspaveli achieved a statistically significant 68% reduction in proteinuria compared to placebo at Week 26, with sustained efficacy through one year in the Phase 3 VALIANT study for C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis.
  • The drug demonstrated consistent efficacy across diverse patient subgroups including adults, adolescents, and those with transplanted kidneys, while maintaining a favorable safety profile with no cases of meningitis or encapsulated bacterial infections.
  • With no currently approved therapies for these rare kidney diseases that affect approximately 15,500 patients in major markets, Aspaveli represents a potential first-in-class treatment option.
  • Sobi and Apellis plan to submit regulatory applications in 2025, with an FDA decision expected by summer 2025 for these devastating conditions that lead to kidney failure in 50% of patients within 5-10 years.

Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

NCT05067127CompletedPhase 3
Apellis Pharmaceuticals, Inc.
Posted 11/12/2021

LEO Pharma Launches Phase 2a Trial of Delgocitinib Cream for Rare Skin Disease Palmoplantar Pustulosis

Rare Disease
  • LEO Pharma has initiated the DELTA NEXT phase 2a trial to evaluate delgocitinib cream versus placebo in adults with mild to severe palmoplantar pustulosis, a rare inflammatory skin condition.
  • The study will recruit up to 135 patients across 40-45 sites in the US, Canada, UK, Germany, and Poland, targeting patients for whom topical corticosteroids are inadequate or inadvisable.
  • Palmoplantar pustulosis currently has no approved advanced systemic treatments in the US or Europe, representing a significant unmet medical need for patients experiencing pain, fatigue, and impaired quality of life.
  • Delgocitinib cream is a topical pan-JAK inhibitor already approved in Europe for chronic hand eczema, offering potential therapeutic benefit through inhibition of multiple inflammatory pathways.

Affinia Therapeutics Partners with DCM Foundation to Advance BAG3 Dilated Cardiomyopathy Awareness and Genetic Testing

Rare Disease
  • Affinia Therapeutics has partnered with the DCM Foundation and Genetic Cardiomyopathy Awareness Consortium to raise awareness about BAG3 dilated cardiomyopathy, a devastating monogenic heart disease affecting over 70,000 patients across the U.S., Europe, and U.K.
  • The collaboration aims to promote early diagnosis and genetic testing for BAG3 DCM, addressing the critical gap where only a fraction of cardiomyopathy patients receive genetic testing despite nearly 50% having a genetic basis.
  • The partnership will establish a BAG3 Patient Advisory Council and educational webinars while advancing Affinia's lead gene therapy candidate AFTX-201, a potential best-in-class AAV therapy delivered as a one-time intravenous injection.
  • Despite current standard of care, almost 25% of BAG3 DCM patients require heart transplant, highlighting the urgent need for innovative treatments and improved diagnostic approaches.

Kamari Pharma Secures $23 Million Series A to Advance First-in-Class TRPV3 Inhibitor for Rare Genetic Skin Diseases

Rare Disease
  • Kamari Pharma closed a $23 million Series A financing co-led by BRM Group and Pontifax to advance KM023, a first-in-class oral TRPV3 inhibitor for rare genetic skin diseases.
  • The lead program KM023 targets three rare genetic skin conditions: Olmsted syndrome, severe keratoderma, and ichthyosis, with proof-of-concept trials planned for each indication.
  • The company plans to initiate a Phase 1b clinical trial for Olmsted syndrome in the second half of 2025, with top-line results expected by year-end.
  • TRPV3 inhibition represents a novel therapeutic approach in rare dermatology, targeting a key regulator in skin disease pathways.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.