This study aims to collect safety and efficacy data during long-term follow-up in SMA patients treated with OAV101 IV or IT. The safety and efficacy of OAV101 will be evaluated up to 15 years after administration.

Start Date30 May 2024

Sponsor / Collaborator

China Novartis Institutes for BioMedical Research Co., Ltd.

[+2]

NCT06019637 / RecruitingNot Applicable

A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study

A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)

Start Date22 Nov 2023

Sponsor / Collaborator

Novartis Pharmaceuticals Corp.

IRCT20221024056284N1 / CompletedNot ApplicableIIT

Evaluation the color match of two single shade resin composites; Charisma Diamond ONE and Charisma Topaz ONE in class five restorations and assessment of dentist and patient satisfaction

Start Date21 Jan 2023

Sponsor / Collaborator

Mashhad University of Medical Sciences

100 Clinical Results associated with Onasemnogene abeparvovec

100 Translational Medicine associated with Onasemnogene abeparvovec

100 Patents (Medical) associated with Onasemnogene abeparvovec

309

Literatures (Medical) associated with Onasemnogene abeparvovec

01 Dec 2024·MUSCLE & NERVE

Outcomes of early‐treated infants with spinal muscular atrophy: A multicenter, retrospective cohort study

Article

Author: Lee, Bo Hoon ; Hamid, Omer Abdul ; Farah, Hiba ; Herbert, Karen ; King, Kiana ; Kuntz, Nancy L. ; Zaidman, Craig M. ; Fisher, Mark ; Gibbons, Melissa ; Lakhotia, Arpita ; Parsons, Julie ; Rehborg, Rebecca ; Rogers, Amanda ; Goedeker, Natalie L. ; Kichula, Elizabeth ; Harmelink, Matthew ; Veerapaniyan, Aravindhan ; Brandsema, John F. ; Felker, Marcia V. ; Arya, Kapil ; Farrar, Michelle A.

Abstract:

Introduction/Aims:

While prompt identification and treatment of infants with spinal muscular atrophy (SMA) can ameliorate outcomes, variability persists. This study assessed management and outcomes of early‐treated infants with SMA.

Methods:

We analyzed retrospective data at 12 centers on infants with SMA treated at age ≤6 weeks from August 2018 to December 2023.

Results:

Sixty‐six patients, 35 with two SMN2 copies and 31 with ≥3 SMN2 copies, were included. Twenty‐five (38%, 22 with two SMN2 copies), had SMA findings before initial treatment which was onasemnogene abeparvovec in 47 (71%) and nusinersen in 19 (29%). Thirty‐two received sequential or combination treatments, including 16 adding nusinersen or risdiplam due to SMA findings following onasemnogene abeparvovec. All sat independently. Compared to children with ≥3 SMN2 copies, those with two SMN2 copies were less likely to walk (23/34 [68%] vs. 31/31 [100%], p < .001) and less likely to walk on time (9/34 [26%] vs. 29/31 [94%], p < .001); one non‐ambulatory child was <18 months old and was excluded from this analysis. No patients required permanent ventilation or exclusively enteral nutrition; six required nocturnal non‐invasive ventilation and four utilized supplemental enteral nutrition, all with two SMN2 copies.

Discussion:

Early treatment of infants with SMA can improve outcomes as indicated by our cohort, all of whom sat independently and are without permanent ventilation. However, our study demonstrates ongoing disability in most children with two SMN2 copies despite early monotherapy and emphasizes the need for additional research, including earlier monotherapy, initial combination therapy, prenatal treatment, and non‐SMN modifying treatments.

01 Dec 2024·Lancet Regional Health-Europe

Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study

Article

Background:

Real-world data on gene addition therapy (GAT) with onasemnogene abeparvovec (OA), including all age groups and with or without symptoms of the disease before treatment are needed to provide families with evidence-based advice and realistic therapeutic goals. Aim of this study is therefore a population-based analysis of all patients with SMA treated with OA across Germany, Austria and Switzerland (D-A-CH).

Methods:

This observational study included individuals with Spinal Muscular Atrophy (SMA) treated with OA in 29 specialized neuromuscular centers in the D-A-CH-region. A standardized data set including WHO gross motor milestones, SMA validated motor assessments, need for nutritional and respiratory support, and adverse events was collected using the SMArtCARE registry and the Swiss-Reg-NMD. Outcome data were analyzed using a prespecified statistical analysis plan including potential predictors such as age at GAT, SMN2 copy number, past treatment, and symptom status.

Findings:

343 individuals with SMA (46% male, 54% female) with a mean age at OA of 14.0 months (range 0-90, IQR 20.0 months) were included in the analysis. 79 (23%) patients were clinically presymptomatic at the time of treatment. 172 (50%) patients received SMN2 splice-modifying drugs prior to GAT (risdiplam: n = 16, nusinersen: n = 154, both: n = 2). Functional motor improvement correlated with lower age at GAT, with the best motor outcome in those younger than 6 weeks, carrying 3 SMN2 copies, and being clinically presymptomatic at time of treatment. The likelihood of requiring ventilation or nutritional support showed a significantly increase with older age at the time of GAT and remained stable thereafter. Pre-treatment had no effect on disease trajectories. Liver-related adverse events occurred significantly less frequently up to 8 months of age. All other adverse events showed an even distribution across all age and weight groups.

Interpretation:

Overall, motor, respiratory, and nutritional outcome were dependent on timing of GAT and initial symptom status. It was best in presymptomatic children treated within the first six weeks of life, but functional motor scores also increased significantly after treatment in all age groups up to 24 months. Additionally, OA was best tolerated when administered at a young age. Our study therefore highlights the need for SMA newborn screening and immediate treatment to achieve the best possible benefit-risk ratio.

Funding:

The SMArtCARE and Swiss-Reg-NMD registries are funded by different sources (see acknowledgements).

01 Nov 2024·MUSCLE & NERVE

Nutrition outcomes of disease modifying therapies in spinal muscular atrophy: A systematic review

Review

Author: O'Brien, Katie ; Woodcock, Ian R. ; Nguo, Kay ; Yiu, Eppie M. ; Billich, Natassja ; Davidson, Zoe E.

Abstract:

The nutritional implications of spinal muscular atrophy (SMA) are profound. Disease modifying therapies (DMT) have improved clinical outcomes. This review describes the impact of DMT on nutrition outcomes. A systematic search strategy was applied across seven databases until May 2023. Eligible studies measured nutrition outcomes in individuals with SMA on DMT (nusinersen, risdiplam or onasemnogene abeparvovec [OA]) compared to untreated comparators. Nutrition outcomes included anthropometry, feeding route, swallowing dysfunction, dietary intake, dietetic intervention, nutritional biochemistry, metabolism, gastrointestinal issues and energy expenditure. Articles retrieved were screened in duplicate, data were extracted and appraised systematically. Sixty three articles from 54 studies were included; 41% (n = 22) investigated nusinersen in pediatric participants with SMA type 1. Anthropometry (n = 18), feeding route (n = 39), and swallowing dysfunction (n = 18) were the most commonly reported outcomes. In combined pediatric and adult cohorts, BMI z‐score remained stable post nusinersen therapy. The proportion of children with SMA requiring enteral nutrition was stable post nusinersen therapy. Ability to thrive at age 1.5 years was higher in children treated in early infancy with OA compared to historical controls. Significant heterogeneity existed across study population characteristics and outcome measures. Nusinersen may prevent deterioration in some nutrition outcomes; and OA in early infancy may be associated with improved nutrition outcomes. Timing of DMT initiation is an important consideration for future nutrition research. Studies investigating nutrition as a primary outcome of DMT, using consistent outcome measures are required for nutritional management strategies for this cohort to be appropriately tailored.

235

News (Medical) associated with Onasemnogene abeparvovec

21 Nov 2024

·www.biopharmadive.com

Novartis wagers more than $1B on gene therapies for the nervous system

Through a new deal, Novartis has added a handful of experimental gene therapies to its neuroscience research pipeline.Novartis is betting the gene therapy technologies developed by Kate Therapeutics, a biotechnology startup founded four years ago, can lead to treatments for an array of neuromuscular diseases. The Swiss pharmaceutical giant said Thursday it has acquired Kate, in a transaction that could ultimately be worth as much as $1.1 billion. Novartis did not disclose what it paid upfront, but a source familiar with the deal told BioPharma Dive the sum was substantially greater than Kates Series A round last year.In buying Kate, Novartis now owns a slate of preclinical gene therapies for rare, muscle-eroding diseases. The biotechs two most advanced programs respectively target Duchenne muscular dystrophy and a condition known as X-linked myotubular myopathy. Astellas Pharma, one of the largest drugmakers is Japan, has been partnering on that latter program since Kate officially launched in June of last year.Kate debuted with $51 million from that Series A fundraising round, which was co-led by its founder, Westlake Village Partners, and Versant Ventures. The company claims its technology which stems from research conducted by a team of scientists at the Broad Institute of MIT and Harvard can make gene therapies that are more precise and able to reach parts of the body they normally have trouble hitting, like the heart. Sharif Tabebordbar, who was the main author of that research, went on to co-found Kate and now serves as its chief scientific officer.We have been highly impressed with the rigor and potential of Kates science, and we are confident this acquisition will further enhance our ability to bring forward new therapeutic options for patients living with neuromuscular diseases, said Fiona Marshall, Novartis president of biomedical research, in a statement.Novartis already has experience in neuromuscular gene therapies, having launched Zolgensma, a blockbuster medicine for spinal muscular atrophy. Zolgensma had been given the growth driver moniker not too long ago, but sales of the drug havent been as strong recently. They totaled $1.2 billion in 2023, down 9% under constant currency exchange rates. And they were flat in the third quarter, at $308 million, compared to the same three-month period last year.Sales from Novartis broader neuroscience business were still up 19% in the third quarter, thanks mostly to the multiple sclerosis medication Kesimpta.The Kate acquisition comes almost a year and a half after Novartis agreed to buy DTx Pharma, a startup aiming to use RNA drugmaking technology to treat neurological diseases. The deal, which carried a $500 million upfront payment, handed Novartis a few experimental drugs that had yet to reach human testing.DTx was one in a series of smaller-scale purchases Novartis made over the past couple years. The company snagged the radiopharmaceutical developer Mariana Oncology for $1 billion, the kidney disease drugmaker Chinook Therapeutics for as much as $3.5 billion, and the cancer specialist Morphosys for roughly $2.9 billion.It also scored a portfolio of potential immune system drugs by buying a subsidiary of IFM Therapeutics for $90 million up front.Ned Pagliarulo contributed reportingEditors note: This story has been updated to further characterize the acquisition price Novartis paid for Kate. '

AcquisitionGene Therapy

18 Nov 2024

·www.globenewswire.com

Trogenix Unveils Revolutionary Platform to Transform Cancer Treatment Through Precision Viral Immunotherapy

Trogenix Unveils Revolutionary Platform to Transform Cancer Treatment Through Precision Viral Immunotherapy Company founded by 4BIO Capital and Professor Steve Pollard as a spinout from the University of Edinburgh to commercialise its breakthrough Synthetic Super-Enhancer platform, Odysseus, to enable unprecedented precision in targeting aggressive cancersIQ Capital, Cancer Research Horizons, National Brain Tumor Society, AIN Ventures and Old College Capital now also investors in Trogenix Dr Ken Macnamara, a veteran gene therapy executive who held leadership roles at Bayer/AskBio and Synpromics, appointed as Chief Executive OfficerTay Salimullah, former executive team member at Novartis Gene Therapies, joins the Board as Trogenix advances towards clinical development Edinburgh, UK, 18 November 2024 -- Trogenix Ltd today emerged from stealth mode to unveil its groundbreaking therapeutic platform for treating aggressive cancers. Built from inception by 4BIO Capital, which specialises in creating breakthrough therapeutic companies, Trogenix spun out from the University of Edinburgh in 2023 and raised a seed financing round earlier this year. Leading life science and deep tech investors IQ Capital, Cancer Research Horizons, the US National Brain Tumor Society’s Brain Tumor Investment Fund, AIN Ventures and the University of Edinburgh’s venture investment fund, Old College Capital, have also invested in Trogenix. "Cancer remains a leading cause of death worldwide, with current therapies often falling short, especially for aggressive solid tumours," said Dr Ken Macnamara, Chief Executive Officer of Trogenix. "We founded Trogenix to transform cancer treatment from chronic disease management to potentially curative one-time treatments. Our Synthetic Super-Enhancer technology represents a significant breakthrough in precision cancer therapy, offering unprecedented selectivity in targeting cancer cells and activating the body's own immune system against tumours, all while leaving healthy cells untouched." The company's lead programme targets glioblastoma (GBM), the most aggressive form of brain cancer, where only 25% of patients survive beyond one year. Preclinical studies have demonstrated curative responses with no toxicity and evidence of persistent anti-tumour immunity, with Phase 1/2 clinical trials planned for 2025. "Trogenix exemplifies our approach to advancing revolutionary therapeutic platforms from concept to reality," said Dmitry (Dima) Kuzmin, Managing Partner at 4BIO Capital and Founding Chairman of Trogenix. "By combining our deep expertise in advanced therapies with the company's breakthrough science, we've helped shape a platform with the potential to fundamentally change cancer treatment. The convergence of synthetic biology, machine learning, gene therapy, and precision targeting in Trogenix's technology represents exactly the kind of innovation needed to deliver next-generation curative treatments. Being able to discern a cancer cell in situ from a healthy cell without relying on a surface antigen is truly revolutionary." The company's proprietary Synthetic Super-Enhancers (SSEs) are engineered DNA elements that act as docking stations for transcription factors uniquely expressed in aggressive cancer cells. This enables highly selective and potent gene control with multiple layers of safety, including direct tumour injection for localised delivery and self-limiting control mechanisms. Through these SSEs, Trogenix's Odysseus platform delivers a comprehensive attack on cancer through controlled cell killing via targeted expression of cytotoxic payloads, immune modulation to overcome tumour-induced immunosuppression, and a unique ‘Trojan Horse’ approach that reawakens the immune system to provide long-term protection against recurrence. World-Class Leadership Team Trogenix emerged from groundbreaking research at the University of Edinburgh - including its UK Centre for Mammalian Synthetic Biology and Institute for Regeneration and Repair - and the Cancer Research UK Scotland Centre. The company was co-founded by Professor Steve Pollard, a renowned authority in regenerative medicine and cancer biology, who serves as Chief Scientific Officer. Under his leadership, the company has rapidly advanced its revolutionary SSE platform from concept to preclinical proof-of-concept. Chief Executive Officer Dr Ken Macnamara brings extensive leadership experience in gene therapy and biotech, including senior roles at AskBio (a Bayer company), and involvement in three successful M&A exits. "Our Odysseus platform represents a significant advancement in cancer treatment," said Professor Steve Pollard, Chief Scientific Officer of Trogenix. "By precisely targeting cancer cell states rather than just cell types, we can achieve unprecedented selectivity while activating the body's own immune system against the tumour. Our preclinical studies in glioblastoma have demonstrated complete responses with no toxicity and evidence of persistent anti-tumour immunity." Beyond GBM, Trogenix is advancing programmes in colorectal cancer liver metastases, hepatocellular carcinoma, lung squamous cell carcinoma, and regenerative medicine, with the goal of submitting five Investigational New Drug (IND) applications in five years. Strategic Board Appointment As Trogenix advances its groundbreaking platform toward clinical development, the company has appointed Tay Salimullah to its Board of Directors, his first Board appointment since leaving Novartis Gene Therapies. Tay has over 20 years of leadership experience in the rare disease and MedTech sectors, with a proven track record in incubating and scaling innovative, high-value technologies, including cell and gene therapies. He spent over a decade at Novartis in a series of leadership roles. As an Executive team member at Novartis Gene Therapies, Tay executed a new commercial model for Zolgensma® securing approvals in more than 55 countries, helping to treat over 4,000 patients, and generating more than $5 billion in revenue. Prior to his roles at Novartis, Tay worked in a variety of investment and healthcare roles, including a decade at Pfizer where he held several strategic and commercial roles. His expertise spans drug development, pricing science, market access, and operational excellence in high-value healthcare solutions. Tay holds a BSc in Management Sciences from the University of Brunel, London. About Trogenix Trogenix unites cutting-edge technologies in genomics, oncology, immunotherapy, and gene therapy to create a revolutionary therapeutic approach through its Odysseus platform. Using proven AAV vectors, our proprietary Synthetic Super-Enhancers (SSEs) are delivered directly to tumour cells without detection. Our SSEs enable unprecedented precision in gene control, effectively revealing cancer to the body's immune system and killing tumour cells. For any cells escaping Trogenix's technology, our 'Trojan horse' reawakens the immune system, countering recurrence and offering potentially curative 'one-and-done' treatments for aggressive tumours. With our lead asset showing curative potential in preclinical glioblastoma studies, we're poised to transform treatment paradigms across multiple cancer types and explore applications beyond oncology, such as regenerative medicine. For more information, visit www.trogenixbio.com. About 4BIO Capital 4BIO Capital (‘4BIO’) is an international venture capital firm headquartered in London, focused solely on the advanced and emerging therapies sector. The 4BIO team, which has an unrivalled network within the advanced therapy sector, comprises leading advanced therapy scientists and experienced life science investors. The firm maintains a global footprint across the US, Europe and Asia with an objective to create, invest in, support, and grow early-stage companies. Its ultimate goal is ensuring access to potentially curative therapies for all patients, with a specific focus on viable, high-quality opportunities in cell and gene therapy, RNA-based therapy, targeted therapies, and the microbiome. 4BIO is currently investing out of its Fund III, having secured support from prominent global institutional investors, including the Children’s Minnesota, UPMC, Development Bank of Japan, Kyowa Kirin, Exor, and many other endowments and foundations. For more information, please visit www.4BioCapital.com For more media enquiries, please contact: trogenix@icrhealthcare.com

Executive ChangeGene TherapyImmunotherapy

11 Nov 2024

·www.globenewswire.com

MediciNova Given Notice of Monetary Damages Due Under Patent Settlement of Sanofi-Novartis

LA JOLLA, Calif., Nov. 11, 2024 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ: MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875), today announced that it was notified by Sanofi that the Sanofi/Novartis litigation was settled. Accordingly, MediciNova is entitled to receive a certain amount of monetary damages through this settlement. Further details will be provided as they become available. Yuichi Iwaki, M.D., Ph.D., MediciNova CEO commented, “The successful resolution of the Sanofi-Novartis litigation and the resulting monetary damages due to MediciNova both validates the value of our intellectual property and provides an additional source of non-dilutive funding, which we plan to apply to supporting our ongoing clinical development programs, particularly our late-stage pipeline targeting inflammatory, metabolic, and neurodegenerative diseases. This outcome further reinforces our commitment to developing unique assets with long-term value. We remain dedicated to preserving our financial position as we continue to prudently advance our novel small molecule therapies towards commercialization." About Sanofi/Novartis litigation On March 30,2022, MediciNova, Inc. was notified that Genzyme Corporation, a subsidiary of Sanofi, filed its First Amended Complaint, which included a claim for infringement of U.S. Patent No. 9,051,542, on February 23, 2022 in a lawsuit previously filed against Novartis Gene Therapies, Inc., Novartis Pharmaceutical Corporation, and Novartis AG in the U.S. District Court for the District of Delaware. This patent, among others was included in the assignment Agreement dated December 19, 2005, between MediciNova, Inc., as successor in interest to Avigen, Inc. and Genzyme Corporation. The plaintiffs allege that the defendants infringe certain U.S. patents through the unauthorized manufacture, use, and sale of recombinant adeno-associated virus vectors (“rAAV vectors”) for their gene therapy drug Zolgensma. If Genzyme recovers any monetary damages, by way of settlement or otherwise, as a result of this litigation, then MediciNova is entitled to receive a certain portion of such monetary damages from Genzyme as per the terms of assignment agreement. About Zolgensma The U.S. Food and Drug Administration (FDA) approved Zolgensma (onasemnogene abeparvovec-xioi) on May 24, 2019. Zolgensma is a gene therapy that treats children under two years old with spinal muscular atrophy (SMA). It was the first gene therapy approved by the FDA to treat a disease. Novartis priced an infusion at $2.1 million, making it one of the priciest medicines on the market. Zolgensma has become one of Novartis’ top-sellers, generating more than $1.3 billion in annual sales in 2023. The company expects it to eventually earn between $1.5 billion and $2 billion annually. Approved in 45 countries, Zolgensma replaces the faulty gene responsible for causing spinal muscular atrophy, a condition that in its severe form robs infants of the ability to stand, sit and, eventually, breathe. Untreated, it’s typically fatal by a very young age. Zolgensma can deliver dramatic benefits, keeping children alive and in some cases helping them achieve motor and developmental milestones they otherwise wouldn’t. In the U.S, its use is limited to babies under the age of two, where it’s seen as having the greatest benefit. About MediciNova MediciNova, Inc. is a clinical-stage biopharmaceutical company developing a broad late-stage pipeline of novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases. Based on two compounds, MN-166 (ibudilast) and MN-001 (tipelukast), with multiple mechanisms of action and strong safety profiles, MediciNova has 11 programs in clinical development. MediciNova’s lead asset, MN-166 (ibudilast), is currently in Phase 3 for amyotrophic lateral sclerosis (ALS) and degenerative cervical myelopathy (DCM) and is Phase 3-ready for progressive multiple sclerosis (MS). MN-166 (ibudilast) is also being evaluated in Phase 2 trials in Long COVID and substance dependence. MN 001 (tipelukast) was evaluated in a Phase 2 trial in idiopathic pulmonary fibrosis (IPF) and a second Phase 2 trial in non-alcoholic fatty liver disease (NAFLD) is ongoing. MediciNova has a strong track record of securing investigator-sponsored clinical trials funded through government grants. Statements in this press release that are not historical in nature constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, without limitation, statements regarding the future development and efficacy of MN-166, MN-001, MN-221, and MN-029. These forward-looking statements may be preceded by, followed by, or otherwise include the words "believes," "expects," "anticipates," "intends," "estimates," "projects," "can," "could," "may," "will," "would," “considering,” “planning” or similar expressions. These forward-looking statements involve a number of risks and uncertainties that may cause actual results or events to differ materially from those expressed or implied by such forward-looking statements. Factors that may cause actual results or events to differ materially from those expressed or implied by these forward-looking statements include, but are not limited to, risks of obtaining future partner or grant funding for development of MN-166, MN-001, MN-221, and MN-029 and risks of raising sufficient capital when needed to fund MediciNova's operations and contribution to clinical development, risks and uncertainties inherent in clinical trials, including the potential cost, expected timing and risks associated with clinical trials designed to meet FDA guidance and the viability of further development considering these factors, product development and commercialization risks, the uncertainty of whether the results of clinical trials will be predictive of results in later stages of product development, the risk of delays or failure to obtain or maintain regulatory approval, risks associated with the reliance on third parties to sponsor and fund clinical trials, risks regarding intellectual property rights in product candidates and the ability to defend and enforce such intellectual property rights, the risk of failure of the third parties upon whom MediciNova relies to conduct its clinical trials and manufacture its product candidates to perform as expected, the risk of increased cost and delays due to delays in the commencement, enrollment, completion or analysis of clinical trials or significant issues regarding the adequacy of clinical trial designs or the execution of clinical trials, and the timing of expected filings with the regulatory authorities, MediciNova's collaborations with third parties, the availability of funds to complete product development plans and MediciNova's ability to obtain third party funding for programs and raise sufficient capital when needed, and the other risks and uncertainties described in MediciNova's filings with the Securities and Exchange Commission, including its annual report on Form 10-K for the year ended December 31, 2023 and its subsequent periodic reports on Form 10-Q and current reports on Form 8-K. Undue reliance should not be placed on these forward-looking statements, which speak only as of the date hereof. MediciNova disclaims any intent or obligation to revise or update these forward-looking statements. INVESTOR CONTACT: David H. Crean, Ph.D.Chief Business OfficerMediciNova, Incinfo@medicinova.com

Phase 2Drug ApprovalPhase 3

100 Deals associated with Onasemnogene abeparvovec

External Link

KEGG	Wiki	ATC	Drug Bank
D11559	Onasemnogene abeparvovec	M09AX	DB15528

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
HMN (Hereditary Motor Neuropathy) Proximal Type I	KR	Novartis Gene Therapies, Inc.	28 May 2021
Spinal Muscular Atrophy	US	Novartis Gene Therapies, Inc.	24 May 2019

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Juvenile Spinal Muscular Atrophy	Phase 3	US	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	JP	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	AU	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	BE	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	CA	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	FR	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	IT	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	TW	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	GB	Novartis Gene Therapies, Inc.	10 Feb 2020
Muscular Atrophy, Spinal, Type II	Phase 3	US	Novartis Gene Therapies, Inc.	10 Feb 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04851873 (SMART, MDA2024) Manual	Phase 3	Spinal Muscular Atrophy	24	onasemnogene abeparvovec	vhetxzqwdb(wfznyldsvb) = strhfdgkoz zfatzbdwlj (urofiwuvhp, 4.0) View more	Positive	04 Mar 2024
NCT05073133 (OFELIA, MDA2024) Manual	Phase 4	Spinal Muscular Atrophy	16	Onasemnogene Abeparvovec	pzkfjatnmn(pwtsuhuhzw) = gtoehscwki euntzlshjm (kiqzdcmpua ) View more	Positive	03 Mar 2024
MDA2024	Not Applicable	-	-	Onasemnogene abeparvovec	nxxezsqvbl(flfflcjgld) = Adverse events were recorded for two patients. None were serious and treatment-related, and event types were consistent with the established safety profile of OA bireikiddc (ktttnuoyig ) View more	-	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Onasemnogene abeparvovec (OA)	xepimpeewo(rgsxvdtqhy) = kztwixovey ntchcvvfxd (orupxijrwe )	-	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Risdiplam (EVRYSDI)	xepimpeewo(rgsxvdtqhy) = feihvewohg ntchcvvfxd (orupxijrwe )	-	03 Mar 2024
RESTORE Registry (MDA2024)	Not Applicable	Spinal Muscular Atrophy	31	Onasemnogene abeparvovec	pgrgilbigy(mssjjttgis) = Two deaths were recorded, neither related to treatment cbxbixphyn (bmkeeemvfx ) View more	Positive	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Onasemnogene abeparvovec	nqwsfytwzm(aunlgnwdhf) = Troponin I elevations observed in OA-treated patients in RESTORE appear to be transient and self-limiting and were not associated with clinically concerning cardiac adverse events xwztmkaviv (zqdldpywjj )	-	03 Mar 2024
SPR1NT (AAN2023)	Phase 3	Spinal Muscular Atrophy SMN2	29	Onasemnogene Abeparvovec	kdjjjsazdr(nnawiwgnyu) = xrzabxgrpy lzegtoguux (nebuywduyi )	-	25 Apr 2023
SPR1NT (AAN2023)	Phase 3	Spinal Muscular Atrophy SMN2	29	Onasemnogene Abeparvovec	kdjjjsazdr(nnawiwgnyu) = cbjxgpdqos lzegtoguux (nebuywduyi )	-	25 Apr 2023
NCT04042025 (LT-002, AAN2023)	Phase 3	Spinal Muscular Atrophy	81	Intravenous onasemnogene abeparvovec	sdswuyhakj(rtxesccabv) = The most frequently reported TEAEs were gastroenteritis, nasopharyngitis, pneumonia, respiratory distress, and viral infection aesvskagmq (freshjcmqq )	Positive	25 Apr 2023
NCT04042025 (LT-002, AAN2023)	Phase 3	Spinal Muscular Atrophy	81	Intrathecal onasemnogene abeparvovec		Positive	25 Apr 2023
AAN2023	Not Applicable	Spinal Muscular Atrophy SMN2	-	Onasemnogene-abeparvovec	yfonivyeth(cosfvtyscw) = phewnbvcbi soumxfvpuq (ptvhbewagu ) View more	-	25 Apr 2023
LT-002 (MDA2023)	Not Applicable	Spinal Muscular Atrophy	81	Intrathecal Onasemnogene Abeparvovec	ykprjqwigf(tztxdpbpqg) = roukwdljxd qolffcbzqi (ftqzajdbkg ) View more	-	19 Mar 2023

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