Gene Therapy

Search documents
X @Bloomberg
Bloomberg· 2025-07-30 02:20
Vinay Prasad, a top regulator at the US Food and Drug Administration, has left the agency after a controversy over his handling of Sarepta's gene therapy https://t.co/QyzJGlMYd0 ...
uniQure(QURE) - 2025 Q2 - Earnings Call Transcript
2025-07-29 13:30
Financial Data and Key Metrics Changes - Revenue for Q2 2025 was $5.3 million, a decrease from $11.1 million in Q2 2024, primarily due to a $7.1 million drop in collaboration revenue and a $2.1 million decrease in contract manufacturing revenue [18][19] - Research and development expenses increased to $35.4 million in Q2 2025 from $33.7 million in the same period in 2024, driven by a $6.3 million rise in external program spending [19] - Cash, cash equivalents, and investment securities totaled $377 million as of June 30, 2025, up from $367.5 million at the end of 2024, attributed to net proceeds of $80.5 million from a follow-on offering [20][21] Business Line Data and Key Metrics Changes - The company is advancing AMT-130 for Huntington's disease, with pivotal data expected in September 2025, and has received breakthrough therapy designation from the FDA [6][7] - AMT-191 for Fabry disease and AMT-260 for mesial temporal lobe epilepsy are also progressing, with initial data from AMT-191 expected at the ICIEM Conference in September [10][17] Market Data and Key Metrics Changes - The company has 14 clinical sites in the U.S. screening patients for AMT-260, with strong interest from the epilepsy community following early positive data [10][17] - There are approximately 35,000 diagnosed Huntington's disease patients in the U.S., with potentially three times that number undiagnosed [70] Company Strategy and Development Direction - The company is focused on preparing for a potential commercial launch of AMT-130 in 2026, with disciplined investments in commercial planning and recruitment for key roles [9][21] - The strategy includes leveraging prior knowledge from Hemgenics to validate the manufacturing process for AMT-130 [12][13] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming pivotal data and regulatory progress, indicating a transformational second half of 2025 [22][24] - The company is committed to delivering transformative therapies for patients with serious unmet needs, emphasizing the urgency of effective treatments for Huntington's disease [23][24] Other Important Information - The company has achieved alignment with the FDA on the statistical analysis plan and CMC requirements for AMT-130, which is crucial for the planned BLA submission [8][12] - The company is actively monitoring the safety and efficacy of AMT-130, including a new cohort for patients with lower striatal volumes [14][59] Q&A Session Summary Question: Does the FDA expect a minimum threshold for clinical benefit versus the enrolled HD on CUHDRS? - Management indicated that the FDA has not requested a minimum clinical effect and is optimistic about the three-year data supporting accelerated approval [26] Question: Can you walk us through the AMT-130 procedure from a patient's perspective? - The procedure is described as minimally invasive, with patients typically recovering quickly and returning to work within days [31][34] Question: What are the expectations for the fourth cohort in the AMT-130 trial? - The goal is to document the safety of administering AMT-130 to patients who would have otherwise been excluded due to lower striatal volumes [58] Question: Are there any differences expected in the regulatory path to approval in Europe versus the U.S.? - The company has not yet met with EMA for scientific advice and is currently focused on the U.S. regulatory path [61] Question: What are the next steps for AMT-260? - The company plans to increase the number of active sites for AMT-260 and is confident in patient enrollment in the second half of the year [59]
uniQure Announces Second Quarter 2025 Financial Results and Highlights of Recent Company Progress
Globenewswire· 2025-07-29 11:05
Core Insights - uniQure N.V. has achieved alignment with the FDA on the statistical analysis plan and CMC requirements for AMT-130, supporting a planned BLA submission in Q1 2026 [1][4] - The company expects to present topline three-year data for AMT-130 in September 2025 [1][4] - Early clinical data for AMT-260 shows a 92% reduction in seizures for the first patient treated, with no serious adverse events reported [1][4] - The appointment of Kylie O'Keefe as Chief Customer and Strategy Officer is aimed at strengthening the leadership team [1][4] - As of June 30, 2025, the company has approximately $377.0 million in cash and equivalents, expected to fund operations into the second half of 2027 [1][5] Recent Developments - The FDA's alignment supports an Accelerated Approval pathway for AMT-130, with a focus on the three-year change in the composite Unified Huntington's Disease Rating Scale [4] - The final statistical analysis plan was submitted to the FDA in July 2025, and a PPQ campaign has been initiated [4] - AMT-130 has received Breakthrough Therapy designation from the FDA based on Phase I/II trial evidence [4] Financial Performance - Revenue for Q2 2025 was $5.3 million, a decrease from $11.1 million in Q2 2024, attributed to changes in license and collaboration revenue [6] - Research and development expenses increased to $35.4 million in Q2 2025 from $33.7 million in Q2 2024, driven by external program spending [9] - Selling, general and administrative expenses decreased to $13.5 million in Q2 2025 from $15.8 million in Q2 2024 [10] - The net loss for Q2 2025 was $37.7 million, compared to a net loss of $56.3 million in the same period in 2024 [13][21] Clinical Programs - AMT-260 is in clinical trials for refractory mesial temporal lobe epilepsy, with initial data showing significant seizure reduction [7] - AMT-191 for Fabry disease and AMT-162 for ALS are also in development, with initial data presentations expected in September 2025 and the first half of 2026, respectively [7] Leadership Changes - Kylie O'Keefe's appointment as Chief Customer and Strategy Officer is part of the company's strategy to enhance its commercial and medical affairs [1][4]
X @Bloomberg
Bloomberg· 2025-07-28 20:54
In a major win for Sarepta, US regulators are recommending that patients who can walk be allowed to take its gene therapy Elevidys again https://t.co/lnGkzEsubE ...
Rocket Refocuses Pipeline As Gene Therapy Sentiment Sours
Benzinga· 2025-07-25 19:02
Core Viewpoint - Rocket Pharmaceuticals is undergoing a strategic reorganization to extend its operational runway and focus on its adeno-associated virus (AAV) cardiovascular platform, which includes several clinical programs [1][2][3]. Group 1: Corporate Reorganization - The restructuring initiative will prioritize resources on clinical programs for Danon disease, PKP2-associated arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy, while also addressing the complete responses to the CRL for KRESLADI [2][3]. - The company plans to reduce its workforce by approximately 30%, which, along with other cost-saving measures, is expected to decrease 12-month operating expenses by nearly 25% [3]. Group 2: Financial Outlook - Rocket anticipates that its existing cash resources will fund operations into the second quarter of 2027 [3]. - Analyst Jason Zemansky from Bank of America downgraded Rocket from Buy to Neutral and reduced the price forecast from $9 to $4, citing weakened investor confidence due to recent failures in the gene therapy sector [5][6]. Group 3: Program Delays and Regulatory Challenges - The company expects delays in the Fanconi Anemia and Pyruvate Kinase Deficiency programs, with regulatory approval for RP-L102 in 2026 now deemed unlikely [4]. - A clinical hold was placed by the FDA on the Phase 2 pivotal trial of RP-A501 for Danon disease, further complicating the company's outlook [4].
Goldman Sachs' Salveen Richter on Sarepta's ongoing troubles, opportunities in biotech
CNBC Television· 2025-07-25 12:10
A new report saying Serepta would need to conduct new studies in order to show the FDA that its musculardrophe drug Elevus is safe. And just this morning, EU regulators not recommending approval of that treatment. As a result, that stock is down another 13%.Joining us right now on that and what to expect from the other biotech names reporting next week is Saline Richtor, lead biotech analyst at Goldman Sachs. and Saline, let's go ahead and start with the news of the day since it's out with Surrepta. Um, jus ...
Solid Biosciences Receives FDA Fast Track Designation for SGT-501 First-in-Class Gene Therapy for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
GlobeNewswire News Room· 2025-07-23 12:00
Core Viewpoint - Solid Biosciences Inc. has received FDA Fast Track, Orphan Drug, and Rare Pediatric Disease designations for SGT-501, a novel gene therapy aimed at treating catecholaminergic polymorphic ventricular tachycardia (CPVT) [1][5][7] Group 1: Product Development - SGT-501 is an AAV-based investigational gene therapy designed to deliver a functional, full-length, codon-optimized copy of the human cardiac calsequestrin (CASQ2) gene to cardiac muscle cells, addressing calcium dysregulation in CPVT [1][5] - The therapy aims to stabilize the ryanodine receptor (RYR2) and normalize cardiac rhythm, potentially preventing life-threatening arrhythmias associated with CPVT [5][8] - A Phase 1b clinical trial is planned to begin in the fourth quarter of 2025, focusing on the safety, tolerability, and efficacy of SGT-501 [3][7] Group 2: Regulatory Designations - The FDA's Fast Track designation is intended to expedite the development and review of drugs for serious conditions that address unmet medical needs, allowing for more frequent interactions with the FDA [2][4] - SGT-501's Fast Track designation reflects the FDA's recognition of the severe unmet needs posed by CPVT, which currently has no FDA-approved therapies [3][4] Group 3: Market Context - CPVT is a rare heart condition affecting approximately 1 in 10,000 individuals globally, often misdiagnosed and primarily identified in children and young adults [8] - The condition is mainly caused by mutations in the RYR2 and CASQ2 genes, leading to impaired calcium regulation in heart muscle cells [8]
Klotho Neurosciences, Inc. Partners with AAVnerGene Inc. to Make Klotho's Gene Therapy Assets
Prnewswire· 2025-07-22 14:21
Core Insights - Klotho Neurosciences, Inc. has partnered with AAVnerGene Inc. to enhance the manufacturing of its gene therapy candidates, aiming for faster, cost-effective, and higher efficacy treatments for neurodegenerative diseases [1][2] - The collaboration focuses on several product candidates, including KLTO-101 for Alzheimer's and Parkinson's diseases, KLTO-202 for ALS, and KLTO-303 for age-related pathologies [2] Company Overview - Klotho Neurosciences, Inc. specializes in innovative cell and gene therapies derived from the human Klotho gene, targeting neurodegenerative and age-related disorders [3] - The company is managed by a team experienced in biopharmaceutical product development and commercialization [3] AAVnerGene Overview - AAVnerGene is a biotechnology company that focuses on next-generation AAV vector technologies, including the AAVone system for cost-effective AAV production and the ATHENA platform for precise tissue targeting [2] - The company aims to provide affordable and scalable gene therapy solutions to accelerate clinical development across various therapeutic areas [2]
Sanofi's Gene Therapy for Eye Disease Gets FDA's Fast Track Tag
ZACKS· 2025-07-16 16:56
Core Insights - Sanofi has received FDA fast-track designation for its gene therapy SAR446597, aimed at treating geographic atrophy (GA) due to age-related macular degeneration (AMD) [1][7] - The fast-track status is designed to expedite the development and review of drugs addressing serious conditions and unmet medical needs [2] - GA affects approximately 1 million people in the U.S. and over 5 million globally, leading to irreversible vision loss [3] Drug Development - SAR446597 is an investigational gene therapy that aims for long-lasting complement inhibition with a single injection, contrasting with current treatments requiring frequent injections [4][7] - The therapy targets two key complement cascade pathways, potentially offering durable treatment for GA [4][5] - Sanofi plans to initiate a phase I/II study to evaluate the safety, tolerability, and efficacy of SAR446597 in GA patients [7][8] Market Context - Apellis Pharmaceuticals' Syfovre is currently the market leader in GA treatment, holding over 60% market share and generating $611.9 million in sales in 2024 [9] - Astellas Pharma's Izervay is also FDA-approved for treating GA secondary to AMD [10] Stock Performance - Year-to-date, Sanofi's shares have declined by 0.2%, while the industry has seen a slight increase of 0.4% [6]
Voyager Adds Fourth Wholly-Owned Alzheimer's Disease Program to Pipeline, Complementing Existing Tau and Amyloid Assets with New APOE Approach
Globenewswire· 2025-07-16 11:00
Core Insights - Voyager Therapeutics is expanding its Alzheimer's disease franchise with a new program targeting apolipoprotein E (APOE), specifically modulating the expression of the high-risk APOE4 variant while delivering the protective APOE2 variant [1][5][6] - The TRACER capsid platform is utilized for intravenous delivery, allowing the bifunctional payload to effectively cross the blood-brain barrier and target relevant brain regions [2][4] - The company aims to leverage its expertise in Alzheimer's biology to advance multiple therapeutic targets, including tau, amyloid, and APOE, to improve patient outcomes [3][5] Company Overview - Voyager Therapeutics is a biotechnology firm focused on using human genetics to treat neurological diseases, with a pipeline that includes programs for Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, and amyotrophic lateral sclerosis (ALS) [5][7] - The company's Alzheimer's disease franchise now includes four wholly-owned assets, including the anti-tau antibody VY7523 and gene therapies targeting tau, amyloid, and APOE [3][5][6] Research and Development - Preclinical studies demonstrated that a single intravenous injection of the TRACER capsid significantly reduced endogenous APOE4 levels while increasing APOE2 expression in relevant brain regions [2][6] - VY7523 is currently in a multiple ascending dose clinical trial, with initial tau PET data expected in the second half of 2026 [6] - VY1706, a tau silencing gene therapy, has shown up to 73% knockdown of tau mRNA in non-human primates and is advancing towards IND in 2026 [6] Technology Platform - The TRACER capsid discovery platform enables rapid identification of novel AAV capsids for gene therapy, facilitating effective delivery across the central nervous system [4][7] - The platform has been validated in cross-species preclinical studies, demonstrating widespread payload expression in the CNS at low doses [4][7]