This study consists of two parts. Part A will evaluate the safety and tolerability of multiple doses of OXB-102 (AXO-Lenti-PD) in participants with Parkinson's disease. Part B will assess the safety and efficacy of the selected dose of OXB-102 in participants with Parkinson's disease.

Start Date17 Oct 2018

Sponsor / Collaborator

Sio Gene Therapies, Inc.

NCT01856439 / TerminatedPhase 1/2

A Multicentre, Open-label Study to Determine the Long Term Safety, Tolerability and Efficacy of ProSavin in Patients With Bilateral, Idiopathic Parkinson's Disease.

The study is designed to assess the long term tolerability of ProSavin and whether it is safe and efficacious in patients administered ProSavin from the PS1/001/07 study .

Start Date01 May 2011

Sponsor / Collaborator

Axovant Sciences, Inc.

[+1]

NCT00627588 / CompletedPhase 1/2

A Phase I/II Study of the Safety, Efficacy and Dose Evaluation of ProSavin®, Administered Using Stereotactic Injection to the Striatum of Patients With Bilateral, Idiopathic Parkinson's Disease.

The primary objectives of the trial are to assess the safety and efficacy of ProSavin.
Patients in the trial will have been diagnosed with Parkinson's disease and will be failing on current treatment with L-DOPA but they will not have progressed to drug-induced dyskinesias. The first stage is an open-label dose escalation to evaluate up to three dose levels of ProSavin in cohorts of three patients each. Following a recommendation by the DMC the study may proceed to the second stage of the trial, a further 12 patients will be recruited to confirm efficacy of the optimal dose in the randomized phase of the study.
The efficacy of ProSavin will be assessed using the Unified Parkinson's Disease Rating Score (UPDRS). Patients will be monitored at regular intervals, with the primary endpoint being an efficacy assessment at six months after treatment. The secondary objective of the trial is to asses the extent to which patients' current therapy (L-DOPA) can be reduced following administration of ProSavin.

Start Date01 Jan 2008

Sponsor / Collaborator

Oxford Biomedica Plc

100 Clinical Results associated with AXO Lenti PD(Oxford Biomedica)

100 Translational Medicine associated with AXO Lenti PD(Oxford Biomedica)

100 Patents (Medical) associated with AXO Lenti PD(Oxford Biomedica)

Literatures (Medical) associated with AXO Lenti PD(Oxford Biomedica)

05 Feb 2023·Cureus

A Review of Genetic and Gene Therapy for Parkinson’s Disease

Review

Author: Dumbhare, Omkar ; Gaurkar, Sagar S

Parkinson's disease (PD) is a syndrome with deterioration of neurons, with its onset starting in the '20s, known as the young beginning of Parkinson's to the late inception of the ailment in the 60s. The majority of the environmental risk associated with PD is age. The pathophysiology of PD is related to the accretion of synuclein alpha (SNCA) protein leading to toxicity. This toxicity further leads to a depletion in dopamine levels, creating both motor and non-motor symptoms. PD is the combination of genetic and environmental risk factors. Linkage and association studies provided data on autosomal dominant and recessive genes linked to PD. Current treatment regimes involve using levodopa, catechol-O-methyl transferase inhibitors, anticholinergics, and monoamine oxidase B (MAO-B) inhibitors. Genetic treatment is done by identifying possible targets. Gene therapy includes silencing, replacing, or correcting the flawed gene with a good gene. This therapy has the advantage of eliminating significant PD symptoms with fewer to no adverse effects than conventional treatment. These targets are organized into disease-modifying or non-disease modifying. The distinction between these two is that disease-modifying treatment stops the degeneration of neurons, while non-disease modifying treatment involves dopaminergic enzyme expression. In non-modifying targets, aromatic L-amino acid decarboxylase (AADC) therapy is used but not as a standalone, so the presentation of AADC, tyrosine hydroxylase (TH), and GTP cyclohydrolase 1 (GCH) is done together as a tricistronic system. With these developments, a drug named prosavin is under clinical phase 1 trial. Disease-modifying targets involve glial cell-derived neurotrophic factor (GDNF). Direct GDNF delivery reduces PD symptoms. This GDNF infusion technique works with a tetracycline-controlled transactivator. Gene therapy introduction into the treatment of PD would be beneficial as there would be lesser adverse effects seen as linked with conventional treatment involving levodopa, MAO-B inhibitors, and anticholinergics, among a few. This article discusses the genetic basis and genetic model of therapy for PD.

01 Sep 2020·Tumour biology : the journal of the International Society for Oncodevelopmental Biology and Medicine

Axolotl Ambystoma mexicanum extract induces cell cycle arrest and differentiation in human acute myeloid leukemia HL-60 cells

Article

Author: Di Fiore, Riccardo ; Suleiman, Sherif ; Schembri-Wismayer, Pierre ; Formosa, Melissa Marie ; Calleja-Agius, Jean ; Cassar, Analisse

Acute myeloid leukemia is the most common form of acute leukemia in adults, constituting about 80% of cases. Although remarkable progress has been made in the therapeutic scenario for patients with acute myeloid leukemia, research and development of new and effective anticancer agents to improve patient outcome and minimize toxicity is needed. In this study, the antitumor activity of axolotl (AXO) Ambystoma mexicanum crude extract was assessed in vitro on the human acute myeloid leukemia HL-60 cell line. The anticancer activity was evaluated in terms of ability to influence proliferative activity, cell viability, cell cycle arrest, and differentiation. Moreover, gene expression analysis was performed to evaluate the genes involved in the regulation of these processes. The AXO crude extract exhibited antiproliferative but not cytotoxic activities on HL-60 cells, with cell cycle arrest in the G0/G1 phase. Furthermore, the AXO-treated HL-60 cells showed an increase in both the percentage of nitroblue tetrazolium positive cells and the expression of CD11b, whereas the proportion of CD14-positive cells did not change, suggesting that extract is able to induce differentiation toward the granulocytic lineage. Finally, the treatment with AXO extract caused upregulation of CEBPA, CEBPB, CEBPE, SPI1, CDKN1A, and CDKN2C, and downregulation of c-MYC. Our data clearly show the potential anticancer activity of Ambystoma mexicanum on HL-60 cells and suggest that it could help develop promising therapeutic agents for the treatment of acute myeloid leukemia.

01 Sep 2019·Molecular therapy. Methods & clinical developmentQ2 · MEDICINE

Gene Therapy for Parkinson’s Disease: Preclinical Evaluation of Optimally Configured TH:CH1 Fusion for Maximal Dopamine Synthesis

Q2 · MEDICINE

ArticleOA

Author: Hosomi, Koichi ; Kelleher, Michelle ; Gipchtein, Pauline ; Robert, Camille ; Palfi, Stéphane ; Mitrophanous, Kyriacos A ; Stewart, Hannah ; Hantraye, Philippe ; Ralph, G Scott ; Fayard, Audrey ; Gourlay, Jeanne ; Van Camp, Nadja ; Binley, Katie ; Badin, Romina Aron ; Jan, Caroline ; Lad, Yatish ; Loader, Julie

A recent phase I-II, open-label trial of ProSavin, a lentiviral vector delivering the key enzymes in the dopamine biosynthetic pathway to non-dopaminergic striatal neurons, demonstrated safety and improved motor function in parkinsonian patients. However, the magnitude of the effect suggested that optimal levels of dopamine replacement may not have been achieved. OXB-102, a lentiviral vector with an optimized expression cassette for dopamine biosynthesis, has been shown to achieve a significantly higher dopamine yield than ProSavin. We assessed the efficacy of OXB-102 in the MPTP macaque model of Parkinson's disease (PD). At 6 months post-vector administration, all treated animals showed significant improvements in clinical scores and spontaneous locomotor activity compared to controls, with the highest recovery observed in the OXB-102 high-dose (HD) group. Positron emission tomography quantification of 6-[¹⁸F]-fluoro-L-m-tyrosine uptake showed a significant increase in amino acid decarboxylase activity for all treated animals, compared with controls, where the OXB-102 HD group showed the highest level of dopaminergic activity. A toxicology study in macaques demonstrated that the vector was safe and well tolerated, with no associated clinical or behavioral abnormalities and no immune response mounted against any transgene products. Overall, these data support the further clinical development of OXB-102 for the treatment of PD.

News (Medical) associated with AXO Lenti PD(Oxford Biomedica)

26 May 2021

·www.globenewswire.com

Global Gene and Cell Therapies Targeting CNS Disorders

Dublin, May 26, 2021 (GLOBE NEWSWIRE) -- The "Gene and Cell Therapies Targeting CNS Disorders - Market Insights and Market Forecast - 2026" report has been added to ResearchAndMarkets.com's offering. This 'Gene and Cell Therapies targeting CNS Disorders - Market Insights and Market Forecast - 2026' report delivers an in-depth understanding of the market trends of Gene and Cell Therapies targeting CNS Disorders in in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan. The Gene and Cell Therapies targeting CNS Disorders market report provides current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted Gene and Cell Therapies targeting CNS Disorders market size from 2018 to 2026, in the 7MM. The report also covers the current Gene and Cell Therapies targeting CNS Disorders treatment practice, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential. Gene and Cell Therapies targeting CNS Disorders emerging drugs NurOwn (BrainStorm Cell Therapeutics) is is mesenchymal stromal stem cells based therapy secreting neurotrophic factors (MSC-NTF) cells. It is designed to target disease pathways in neurodegenerative disorders by effectively delivering NTFs directly to the site of damage. MSC-NTF cells are generated using autologous, mesenchymal stem cells (MSCs) that have been extracted from bone marrow, expanded and differentiated ex-vivo. The therapy is currently under clinical evaluation in phase III for the treatment of Amyotrophic lateral sclerosis (ALS) and in phase II for treatment of Multiple Sclerosis, Chronic Progressive. Neuro-Cells (Neuroplast) is an autologous cell preparation that aims to decrease inflammation and reduce cell death. Neuro- Cells is produced in a certified and patented closed system manufacturing process from bone marrow and is a registered Advanced Therapy Medicinal Product (ATMP). Based on these mechanisms, Neuroplast focuses on developing a stem cell therapy, which can inhibit the inflammatory processes following damaged/dying neural tissue cells and sees its Neuro-Cells as a disease-modifying therapy (DMT). Currently it is being evaluated in phase I and phase II/III clinical studies in treatment of Spinal Chord Injuries (SCI). Gene and Cell Therapies targeting CNS Disorders Drugs Uptake This section focuses on the rate of uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2018-2026. The analysis covers Gene and Cell Therapies targeting CNS Disorders market uptake by drugs, patient uptake by therapies, and sales of each drug. This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and allow the comparison of the drugs based on market share and size, which again will be useful in investigating factors important in the market uptake and in making financial and regulatory decisions. Gene and Cell Therapies targeting CNS Disorders Pipeline Development Activities The report provides insights into different therapeutic candidates in Phase III, Phase II/III, Phase II, and Phase I/II stage. It also analyses Gene and Cell Therapies targeting CNS Disorders' key players involved in developing targeted therapeutics. Pipeline Development Activities The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for Gene and Cell Therapies targeting CNS Disorders emerging therapies. Key Topics Covered: 1. Key Insights2. Report Introduction3. Executive Summary4. Gene and Cell Therapies targeting CNS disorders: Overview4.1. Introduction to CNS Disorders4.2. Role of Cell and Gene Therapy in CNS Disorders4.3. Multiple Sclerosis4.4. Parkinson's disease4.5. Spinal cord injury4.6. Amyotrophic Lateral Sclerosis4.7. Huntington's disease4.8. Alzheimer's disease4.9. Spinal Muscular Atrophy5. Landscape Assessment5.1. Regulatory Scenario of Cell and Gene Therapies in the 7MM5.2. United States5.3. The European Union5.3.1. United Kingdom5.3.2. Germany5.3.3. France5.3.4. Italy5.3.5. Spain5.4. Japan5.5. Reimbursement Scenario of Cell and Gene Therapies in the 7MM5.5.1. United States5.5.2. United Kingdom5.5.3. Germany5.5.4. France5.5.5. Italy5.5.6. Spain5.5.7. Japan5.6. Mergers and Acquisitions5.7. Industry News and Policies by Regions5.7.1. Industry News5.7.2. Policies and Government Initiatives for Cell and Gene Therapies6. Marketed Products6.1. Zolgensma: Novartis7. Emerging Therapies for Amyotrophic Lateral Sclerosis7.1. NurOwn: BrainStorm Cell Therapeutics7.2. Engensis: Helixmith7.3. HYNR-CS inj: Corestem7.4. Q Cells: Q therapeutics7.5. VM202: Helixmith7.6. RAPA-501 Autologous T cells: Rapa Therapeutics8. Emerging Therapy for Multiple sclerosis8.1. NurOwn: BrainStorm Cell Therapeutics8.1.1. Product Description8.1.2. Product Developmental Activities9. Emerging Therapy for Spinal Cord Injury9.1. Neuro-Cells: Neuroplast9.2. Umbilical Cord Blood Mononuclear Cell: StemCyte9.3. FAB117-HC: Ferrer Internacional9.4. Human spinal cord stem cells: Neuralstem9.5. FAB117-HC: Ferrer Internacional10. Emerging therapies for Alzehimer10.1. itMSCs: Stemedica Cell Technologies10.2. AAV-hTERT: Libella Gene Therapeutics10.3. ST-501: Sangamo Therapeutics11. Emerging Therapies for Parkinson Disease11.1. VY-AADC: Hoffmann-La Roche11.2. Lomecel-B: Longeveron11.3. OXB-102: Sio Gene Therapies11.4. PR001: Prevail Therapeutics (Eli Lilly and Company)11.5. Neural Stem Cell-Derived Neurons: NeuroGeneration11.6. AAV2-GDNF: Brain Neurotherapy Bio (AskBio)12. Emerging Therapy for Huntington12.1. Intra-striatal rAAV5-miHTT: UniQure Biopharma12.1.1. Product Description12.1.2. Research and Development12.1.3. Product Developmental Activities13. Company Profiles For more information about this report visit

Gene TherapyCell TherapyAcquisitionCollaborate

19 Nov 2008

·www.biospace.com

Oxford BioMedica PLC Announces Six-Month Efficacy Results With Low Dose of ProSavin(R) in Phase I/II Trial in Parkinson’s Disease

Oxford, UK – 19 November 2008: Oxford BioMedica (LSE: OXB), a leading gene therapy company, announced today promising additional results from the low-dose cohort of patients in the Phase I/II trial of its novel gene therapy, ProSavin, for the treatment of Parkinson’s disease. The three patients in this cohort showed improved motor function, as measured by the Unified Parkinson’s Disease Rating Score (UPDRS) in the ‘off’ state, of an average of 30% at six months. The robust safety pro ProSavin has been maintained at six months with no evidence of adverse events or immunologic reactions to the treatment. In addition, all patients showed improvement in Quality of Life, as measured by the industry-standard PDQ-39 questionnaire, at six months. The three patients that received the low dose of ProSavin demonstrated improvements in their UPDRS motor ‘off’ scores in the range of 10-50% after six months. This score measures the degree of mobility in the absence of standard of care dopaminergic therapies. One patient showed an improvement of up to 50% at six months, which was an increase from the three-month assessment, and another patient maintained an improvement of 30%. One patient showed an improvement of 10% at the six-month assessment compared to 23% at three months, although this patient’s score may have been affected by adjustments to his L-DOPA ‘equivalent’ therapy. The clinical evaluation of the high dose of ProSavin is progressing. There have been no safety issues to date and the preliminary data from the first patient treated at the high dose are promising. The principal investigator for the trial, Professor Stéphane Palfi from the Henri Mondor Hospital in Paris, will include the six-month data in his presentation at the 38th Annual Meeting of the Society for Neuroscience in Washington DC, USA. The presentation will be given at 3.00pm (EST) on Wednesday, 19 November (session: Parkinson’s Disease Interventions in Animal Models and Humans; presentation title: A Phase I/II clinical trial for Parkinson’s disease using ProSavin). Professor Palfi commented on the new data: "It is very encouraging that the early trend in benefit with the low dose of ProSavin has been maintained at six months. If the higher levels of efficacy are confirmed as the trial progresses, ProSavin would represent a fundamental new treatment option for patients with Parkinson’s disease." John Dawson, Chief Executive Officer of Oxford BioMedica, added: "We are delighted by the progress of the Phase I/II trial of ProSavin. We look forward to reporting data from the high-dose group in the first half of 2009. Given the potential opportunity for ProSavin to address the unmet need in Parkinson’s disease, we are advancing our discussions with potential partners with the aim of accelerating development and commercialisation of the product."

CollaborateGene Therapy

100 Deals associated with AXO Lenti PD(Oxford Biomedica)

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Parkinson Disease	Phase 2	FR	Oxford Biomedica Plc	01 Jan 2008
Parkinson Disease	Phase 2	GB	Oxford Biomedica Plc	01 Jan 2008

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03720418 (SUNRISE-PD, MDS2021)	Phase 1/2	Parkinson Disease	4	ALPD low dose (4.2E+6 TU)	(zxklariqpg) = One subject had two serious adverse events of confusional state and wound infection which resolved and were unrelated to ALPD and possibly related to the surgical procedure kzkeiiduwn (cbsvfdlbot ) View more	-	17 Sep 2021
				ALPD mid dose (1.4E+7 TU)
NCT03720418 (GlobeNewswire) Manual	Phase 2	Parkinson Disease	4	AXO-Lenti-PD Gene Therapy	ihzragahmv(zjxvyboszh) = njunxyemqn sakaxdugzj (kzpnagdhnn ) View more	Positive	06 Oct 2020
ASGCT2014	Not Applicable	Parkinson Disease tyrosine hydroxylase \| aromatic L-amino acid decarboxylase \| GTP-cyclohydrolase	-	OXB-102 full-strength dose	ptpfmepkmk(ntbimjwpnc) = rnnrggggho olthmefosh (hnpzrsbtrs ) View more	Positive	01 May 2014
				OXB-102 1/5th dose	ptpfmepkmk(ntbimjwpnc) = ivdlzrshug olthmefosh (hnpzrsbtrs ) View more

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AXO Lenti PD(Oxford Biomedica)

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