Neurogene
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- -
- Employees
- 91
- Market Cap
- $466.9M
- Introduction
Neurogene, Inc. is a clinical-stage biotechnology company, which engages in the development of product portfolio of genetic medicines for rare neurological diseases. The company was founded in May 2007 and is headquartered in New York, NY.
Neurogene Unveils Protocol to Reverse HLH Syndrome Associated with High-Dose AAV Gene Therapy
• Neurogene has developed a monitoring and treatment algorithm designed to reverse hemophagocytic lymphohistiocytosis (HLH), a rare hyperinflammatory syndrome linked to high-dose AAV gene therapy.
• The protocol focuses on daily monitoring of the "three Fs" - elevated ferritin levels, fever, and falling blood counts - with high-dose corticosteroids as first-line treatment and anakinra as second-line therapy.
• Despite using a lower dose (E13 vg/kg range) in its Phase 1/2 trial of NGN-401 for Rett syndrome, Neurogene has incorporated this protocol as a precautionary measure, as HLH has not been reported at this dose level.
CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder
• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder.
• The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein.
• This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.
Shionogi to Acquire Japan Tobacco's Pharmaceutical Subsidiaries for $1.1 Billion
• Shionogi & Co. plans to acquire Japan Tobacco's pharmaceutical subsidiaries Torii Pharmaceutical and Akros Pharma for ¥160 billion ($1.1 billion) to strengthen its global R&D capabilities.
• The acquisition, negotiated since early 2024, will proceed through a tender offer beginning June 18 with a share price of ¥6,350 to acquire at least 11.89% of the minority stake.
• This strategic move represents significant consolidation in the Japanese pharmaceutical sector and expands Shionogi's market presence amid increasing global competition.
Neurogene Halts High-Dose Arm of Rett Syndrome Gene Therapy Trial Following Serious Adverse Event
• Neurogene has suspended enrollment in the high-dose arm of its NGN-401 gene therapy trial for Rett syndrome after a patient developed life-threatening systemic hyperinflammatory syndrome, a known risk with AAV vector therapies.
• Despite early efficacy signals showing "meaningful gains of skills and developmental milestones" in low-dose patients, the company's shares plummeted nearly 44% following the safety concerns announcement.
• The trial will continue with the low-dose cohort, though with delayed enrollment timeline, as the company works to balance promising efficacy data against serious safety considerations for this devastating neurological disorder.
FDA's START Program Selects First Rare Disease Therapies for Accelerated Development
• The FDA's START program, designed to expedite rare disease therapy development, has chosen its first candidates for enhanced guidance and support.
• Denali Therapeutics' DNL12 for mucopolysaccharidosis IIIA, Neurogene's NGN-401 for Rett syndrome, and Larimar Therapeutics' nomlabofusp for Friedreich's ataxia are among the selected therapies.
• Grace Science's GS-100 gene therapy for NGLY1 deficiency is also included, marking a significant step forward in addressing this life-threatening condition with no approved treatments.
• The START program aims to provide comprehensive support in clinical trial design and data generation, facilitating efficient development of potentially life-saving therapies.
Sarepta Seeks Accelerated Approval for DMD Gene Therapy SRP-9001
• Sarepta Therapeutics has submitted SRP-9001 (delandistrogene moxeparvovec) to the FDA for accelerated approval to treat ambulatory Duchenne muscular dystrophy (DMD) patients.
• The filing is based on positive data from early-stage studies, showing improvements in clinical function and a consistent safety profile, while awaiting Phase 3 EMBARK results.
• SRP-9001, a one-time gene therapy, delivers a shortened dystrophin gene via an AAV vector, addressing the underlying genetic defect in DMD patients.
• If approved, SRP-9001 would offer a one-time treatment option for DMD, contrasting with Sarepta's existing chronic exon-skipping therapies.
Breakthroughs and Challenges in Gene Therapy: A 2024 Overview
2024 has seen significant advancements and challenges in the field of gene therapy, including promising results from Novartis' spinal muscular atrophy treatment, India's first successful gene therapy for haemophilia, and the development of a new prenatal gene therapy technique. However, the year also highlighted the risks associated with gene therapy, as seen in the death of a clinical trial participant and the development of blood cancer in children treated with Bluebird bio's Skysona.
Key Advances in Neuromuscular and Movement Disorder Treatments Highlighted in 2024
• AbbVie's Vyalev, a 24-hour subcutaneous levodopa infusion, gained FDA approval for managing motor fluctuations in advanced Parkinson's, offering a novel therapeutic approach.
• Intellia's CRISPR therapy, NTLA-2001, demonstrated safe redosing in ATTR amyloidosis patients, achieving additive pharmacodynamic effects on the target protein.
• A phase 3 study revealed that buntanetap is a safe and well-tolerated drug which improves motor, nonmotor, and cognitive symptoms of Parkinson's disease.
• The FDA supported using αSyn-SAA biomarker in Parkinson's clinical trials, enhancing therapeutic development through improved diagnostic precision.
Gene Therapies Show Promise in Targeting the Root Cause of Rett Syndrome
• Two companies, Neurogene and Taysha Gene Therapies, are in Phase 1/2 clinical trials with gene therapies targeting the MECP2 gene for Rett syndrome.
• These therapies aim to address the underlying genetic cause of Rett syndrome, unlike existing treatments that only manage symptoms.
• Both companies have incorporated regulatory mechanisms to ensure precise MECP2 expression, avoiding complications from overexpression.
• Preliminary data from ongoing trials are expected in late 2024 and early 2025, offering hope for a potential disease-modifying treatment.
FDA Investigates Blood Cancer Risk Linked to Bluebird Bio's Skysona Gene Therapy
• The FDA is investigating Bluebird Bio's Skysona gene therapy due to reports of hematologic malignancies, including myelodysplastic syndrome and acute myeloid leukemia.
• These adverse events were observed in clinical trials, with diagnoses occurring 14 to 92 months post-treatment, raising concerns about long-term safety.
• The FDA advises healthcare providers to consider alternative treatments like allogeneic stem cell transplants before using Skysona, especially for patients with suitable donors.
• Patients treated with Skysona should undergo lifelong monitoring for malignancies, including regular blood counts and bone marrow evaluations.
Solengepras Enters Phase 3 for Parkinson's, SAGE-718 Discontinued, and Neurogene Pauses NGN-401 High-Dose Trial
• Cerevance's solengepras (CVN424) has entered a Phase 3 trial (ARISE) as an adjunctive therapy for Parkinson's disease, with topline data expected in the first half of 2026.
• Sage Therapeutics discontinued the development of SAGE-718 (dalzanemdor) for Huntington's disease after disappointing Phase 2 results showing no significant cognitive improvement.
• Neurogene paused the high-dose arm of its Phase 1 study of NGN-401, a gene therapy for Rett syndrome, following a serious adverse event in one participant.
Advancements in Gene and Cell Therapies Target Diverse Diseases
• Ultragenyx seeks accelerated FDA approval for UX111, a gene therapy for MPSIII, based on Phase 1/2/3 trial data.
• Arbor Biotechnologies' CRISPR-based therapy ABO-101 receives clearance for US trial in Primary Hyperoxaluria Type 1.
• Allogene Therapeutics' ALLO-329 cleared by FDA for Phase 1 trial in rheumatology indications including lupus.
• uniQure progresses in trial for SOD1-ALS gene therapy AMT-162, advancing to the second cohort enrollment.
Neurogene Halts High-Dose Arm of Rett Syndrome Trial After Patient Death
• Neurogene's Phase I/II Rett syndrome trial experienced a patient death linked to the higher dose (3E15 vg) of NGN-401 gene therapy, triggering systemic hyperinflammatory syndrome.
• The FDA has permitted Neurogene to continue the trial, but only with the lower dose (1E15 vg) of NGN-401, which has shown some clinical benefits in early data.
• Neurogene plans to provide further details on registrational trial plans for NGN-401 in the first half of 2025, with more Phase I/II data expected later next year.
• The company has also suspended its Batten disease program after failing to secure Regenerative Medicine Advanced Therapy designation from the FDA.
Neurogene's Rett Syndrome Gene Therapy Trial Pauses High Dose After Patient Death
• A patient with Rett syndrome died after receiving a high dose of Neurogene's experimental gene therapy, NGN-401, triggering a safety review.
• The FDA is allowing Neurogene to continue the Phase 1/2 clinical trial with a lower dose of NGN-401, which has shown promising early results.
• The adverse event underscores the risks associated with high doses of AAV vectors in gene therapy, particularly regarding immune responses.
• Neurogene plans to update the clinical trial protocol to remove the high dose and expects to resume dosing in the low-dose cohort after revisions.
Neurogene's Rett Syndrome Trial Halted After Patient Death
• A patient in Neurogene's Phase I/II trial for Rett syndrome gene therapy, NGN-401, died due to complications from a hyperinflammatory syndrome linked to AAV-based therapies.
• The patient received a 3E15 vg dose of NGN-401 on November 5th, and the trial has been paused for the 3E15 vg dose, with no further participants to be enrolled in that arm.
• Neurogene's stock price has significantly decreased following the adverse event and subsequent death, impacting the company's market value.
• The company plans to engage with the FDA to plan a future registrational clinical trial and expects to provide an update on the trial design in the first half of 2025.
Halozyme Abandons Evotec Acquisition, Neurogene Trial Faces Setback, Argenx Advances Myositis Program, and Kyowa Kirin Licenses Kura Oncology's Leukemia Drug
• Halozyme withdrew its $2.1 billion offer to acquire Evotec after the latter declined to engage in acquisition talks, despite Halozyme's repeated attempts to initiate discussions.
• A patient in Neurogene's Rett syndrome gene therapy trial died due to complications from a hyperinflammatory syndrome, a known risk associated with AAV-delivered gene therapies.
• Argenx is advancing its efgartigimod program into Phase 2/3 trials for myositis subtypes after positive Phase 2 results showing significant patient improvement compared to placebo.
• Kyowa Kirin is paying Kura Oncology $330 million upfront for rights to ziftomenib, an experimental oral leukemia drug, with potential for $420 million more upon market approval.
Gene Therapy Advances: From FDA Clearances to Clinical Trial Progress in Early 2025
• The FDA cleared United Therapeutics' UKidney for US trials, a gene-edited porcine kidney product intended for end-stage renal disease via xenotransplant.
• uniQure received the green light to proceed with the second dose cohort in their Phase 1/2 trial of AMT-162 for SOD1-ALS gene therapy.
• Sarepta Therapeutics' Elevidys showed sustained benefit in ambulatory patients with Duchenne muscular dystrophy in Phase 3 trial results.
Neurogene Pauses High-Dose NGN-401 Trial for Rett Syndrome After Serious Adverse Event
• Neurogene has paused the high-dose arm of its Phase 1/2 trial of NGN-401, a gene therapy for Rett syndrome, following a serious adverse event in one participant.
• The affected participant in the high-dose cohort developed systemic hyperinflammatory syndrome and is in critical condition, prompting a safety review.
• The FDA has allowed continued dosing in the low-dose cohort, where treatment-related adverse events have been mild and responsive to steroids.
• Despite the setback, earlier data from the low-dose cohort showed improvements in key Rett syndrome symptoms, with plans for a Phase 3 trial still under consideration.
FDA Rejects Astellas' Izervay Update; Cytokinetics and Bayer Announce Aficamten Licensing Deal
• The FDA rejected Astellas' request to update Izervay's prescribing information for bi-monthly injections due to a statistical matter, maintaining Apellis' dosing advantage.
• Cytokinetics granted Bayer exclusive rights to develop and commercialize aficamten in Japan, with potential milestone payments for Cytokinetics.
• Jupiter Bioventures, founded by Norman Sharpless and Nathaniel David, launched with $70 million to create startups focused on cancer and other diseases.
© Copyright 2025. All Rights Reserved by MedPath