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Ascidian Therapeutics Advances RNA Exon Editing Platform with Key Leadership Appointments and Clinical Progress

Rare Disease
  • Ascidian Therapeutics has assembled a distinguished Scientific Advisory Board featuring globally recognized RNA biology experts to guide its RNA exon editing platform development.
  • The company appointed Dr. Murray Abramson as Chief Development Officer, bringing over 25 years of clinical development experience including successful filings of more than 6 NDAs and BLAs.
  • ACDN-01, the first RNA exon editor in clinical development, is currently being evaluated in the Phase 1/2 STELLAR trial for Stargardt disease, representing the most advanced clinical program targeting the underlying cause of this inherited retinal disorder.
  • The company's RNA exon editing technology enables precise, kilobase-scale editing of RNA in vivo without foreign enzymes or genomic alterations, potentially addressing diseases that have eluded conventional gene therapies.

Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

NCT06467344RecruitingPhase 1
Ascidian Therapeutics, Inc
Posted 6/11/2024

Vivo Capital Secures $740 Million for Third Cycle of Healthcare Investment Fund

Drug ApprovalRare DiseaseOncology
• Vivo Capital has closed the third cycle of its Opportunity Fund with over $740 million in commitments, focusing on preclinical and clinical-stage life sciences companies developing novel therapies.
• The fund has previously backed companies that achieved significant milestones, including FDA approvals for treatments targeting COPD, myelodysplastic syndrome, narcolepsy, and Prader-Willi Syndrome.
• Several Vivo-backed companies have been acquired by pharmaceutical giants in billion-dollar deals, including RayzeBio (Bristol-Myers Squibb, $4.1B), Chinook Therapeutics (Novartis, $3.2B), and Sierra Oncology (GSK, $1.9B).

Dawn Health Secures €11.5 Million to Expand Pharma-Focused Digital Health Platform

AIReal World EvidenceOncologyRare Disease
  • Copenhagen-based Dawn Health has raised €11.5 million from existing investors to scale its regulatory-grade digital health platform designed specifically for pharmaceutical companies.
  • The platform, already utilized by industry leaders like Merck and Novartis, supports patients with conditions including oncology, multiple sclerosis, and rare pediatric diseases through AI-driven symptom tracking and clinical integration.
  • This funding will accelerate Dawn Health's SaaS delivery model, product development, and international expansion as the company aims to become the global leader in pharmaceutical digital health solutions.

Neurofibromatosis Type 1 with Plexiform Neurofibromas: Diagnosis, Epidemiology and Clinical Manifestations

Rare DiseaseNeurology
  • Neurofibromatosis Type 1 (NF1) affects approximately 1 in 3000 individuals globally, with plexiform neurofibromas (PNs) occurring in up to 50% of NF1 patients.
  • NF1 is typically diagnosed in early childhood between ages 4-6 using NIH clinical criteria, including café-au-lait macules, neurofibromas, and other characteristic manifestations.
  • Plexiform neurofibromas are usually congenital but become clinically apparent in early childhood, with symptoms including disfiguring masses, pain, neurological deficits, and compression of adjacent structures.

FDA Approves Miplyffa for Niemann-Pick Type C Disease, Offering New Hope for Rare Genetic Disorder

Drug ApprovalRare DiseaseOrphan DrugFDA Approval
  • The FDA has approved Miplyffa, a new medication for treating Niemann-Pick Type C (NPC), a fatal genetic disorder affecting approximately 900 people in the United States.
  • Miplyffa works by protecting cells from fat accumulation and slowing disease progression, helping patients maintain neurological function for longer periods.
  • The drug is approved for children over two years old and has shown promising results in early patients, with some experiencing stabilization or improvement in disease markers within three months.
  • NPC is a progressive disorder that typically robs patients of their ability to speak, think clearly, swallow, walk, and move, often affecting children and young adults.

Lundbeck Presents Phase II Data for Amlenetug in Multiple System Atrophy at International Congress

Monoclonal AntibodyRare Disease
  • Lundbeck presented results from the AMULET phase II trial investigating amlenetug, a monoclonal antibody targeting α-synuclein, as a potential treatment for multiple system atrophy (MSA).
  • The company shared new insights from the TALISMAN natural history study, providing critical data on early disease progression in MSA patients to support phase III development.
  • Patient perspectives from the AMULET trial informed the design of the upcoming phase III MASCOT trial, demonstrating Lundbeck's commitment to patient-centered drug development.
  • MSA remains a rare, rapidly progressing neurodegenerative disease with no approved therapies and significant unmet medical need.

A Study of Lu AF82422 in Participants With Multiple System Atrophy

NCT05104476Active, Not RecruitingPhase 2
H. Lundbeck A/S
Posted 11/16/2021

A Trial of Lu AF82422 in Participants With Multiple System Atrophy (MSA)

NCT06706622RecruitingPhase 3
H. Lundbeck A/S
Posted 12/3/2024

Rett Syndrome Pipeline Analysis Reveals 20+ Companies Developing Novel Therapeutics with Gene Therapy Leading Innovation

Rare DiseaseNeurology
  • A comprehensive 2025 pipeline analysis identifies over 20 companies developing more than 20 therapeutic candidates for Rett syndrome, highlighting significant industry investment in this rare neurological disorder.
  • Anavex Life Sciences leads with blarcamesine in Phase III trials, representing the most advanced treatment targeting SIGMA1 and muscarinic receptors to restore cellular homeostasis.
  • Gene therapy approaches dominate the pipeline with TSHA-102 and NGN-401 delivering MECP2 gene replacement using AAV9 vectors in Phase I/II trials.
  • The therapeutic landscape spans multiple modalities including small molecules, gene therapies, and biologics, addressing the critical unmet medical need in Rett syndrome treatment.

CRISPR Therapeutics' Casgevy Shows Commercial Progress as Gene Therapy Pipeline Advances

Rare Disease
  • CRISPR Therapeutics and Vertex report encouraging progress with Casgevy, their approved gene therapy for sickle cell disease and beta-thalassemia, with 65 treatment centers now activated globally.
  • Early clinical data for CTX310, CRISPR's in vivo liver-editing therapy, demonstrates significant reductions in LDL cholesterol and triglycerides, showing promise for cardiovascular applications.
  • Despite gene therapy's commercial challenges, Vertex executives believe Casgevy has "multibillion-dollar" potential, with 90 patients having undergone initial cell collection and eight completing the full treatment process.

TuHURA Biosciences Initiates Phase 1b/2a Trial of IFx-Hu2.0 for Deep-Seated Merkel Cell Carcinoma

OncologyRare Disease
  • TuHURA Biosciences has launched a phase 1b/2a trial evaluating IFx-Hu2.0 combined with pembrolizumab in patients with noncutaneous Merkel cell carcinoma of unknown primary origin.
  • The study targets up to 30% of MCC patients who present without primary skin lesions, focusing on deep-seated tumors in liver, lungs, or retroperitoneum.
  • IFx-Hu2.0 is designed to overcome primary resistance to checkpoint inhibitors and has shown systemic antitumor immune responses in previous trials.
  • Initial data from the 9-patient multicenter trial are expected in late 2025 or early 2026, with potential expansion to other cancer types if successful.

IFx-Hu2.0 As An Adjunctive Therapy To Pembrolizumab In Advanced Or Metastatic Merkel Cell Carcinoma (MCC)

NCT06940440RecruitingPhase 1
TuHURA Biosciences, Inc.
Posted 10/1/2025

Placebo-Controlled Trial of IFx-Hu2.0 Followed By Pembrolizumab In Checkpoint Inhibitor Naïve Participants With Advanced Or Metastatic Merkel Cell Carcinoma

NCT06947928RecruitingPhase 2
TuHURA Biosciences, Inc.
Posted 10/1/2025

Vigil Neuroscience Advances Dual TREM2 Programs with Promising Results for Neurodegenerative Diseases

Rare DiseaseMonoclonal Antibody
  • Vigil Neuroscience is on track to report final analysis from the IGNITE Phase 2 trial of iluzanebart for adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) in Q2 2025, with plans to pursue accelerated approval.
  • The company's small molecule TREM2 agonist VG-3927 demonstrated positive Phase 1 results for Alzheimer's disease, showing favorable safety, CNS penetration, and robust target engagement, with Phase 2 trials planned for Q3 2025.
  • Preclinical data presented at the AD/PD™ 2025 conference highlighted VG-3927's unique synergistic activation with endogenous TREM2 ligands like amyloid-beta, potentially enhancing potency in regions of pathology.

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