Continued Evaluation of Patients With Parkinson's Disease Who Previously Received BRT-DA01, a Human Embryonic Stem Cell-Derived Midbrain Dopaminergic Neuronal Cell Therapy

This is a multicenter study to evaluate the safety and clinical outcomes of BRT-DA01 in subjects with PD who previously received BRT-DA01 in the Phase 1 Study MSK-DA01-101.No investigational therapy will be administered in this study.

开始日期2023-05-16

申办/合作机构

BlueRock Therapeutics LP

NCT04802733 / Completed临床1期

Phase 1 Study To Assess the Safety and Tolerability of Human Embryonic Stem Cell-Derived Midbrain Dopamine Neuron Cell Therapy (MSK-DA01) For Advanced Parkinson's Disease

This clinical trial is designed to test whether surgically injecting nerve cells that make dopamine into the brain of Parkinson's disease patients is safe, and to monitor for potential side effects.

开始日期2021-05-03

申办/合作机构

BlueRock Therapeutics LP

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2024-10-01·JOURNAL OF ETHNOPHARMACOLOGY

Anti-arthritic potential and antioxidant properties of infusion, fractions and flavonoid glycosides from Dipteryx alata (baru) leaves

Article

作者: de Matos Balsalobre, Natalia ; Maurino dos Santos, Jéssica ; Fava de Souza, Maiara ; Kappel Trichez, Virginia Demarchi ; Mussury Franco da Silva, Rosilda Mara ; Nazari Formagio, Anelise Samara ; Marangoni Faoro, Janaine Alberto ; Leite Kassuya, Candida Aparecida ; Maurino Dos Santos, Jéssica

ETHNOPHARMACOLOGICAL RELEVANCE:

Dipteryx alata Vogel., popularly known as "baru", is a native species of Brazilian cerrado used by "Ribeirinhos" in the North Araguaia microregion. In the traditional medicine, maceration of barks or leaves infusion are used to treat back and muscle pain, osteoporosis and rheumatism. However, few studies have demonstrated the pharmacological effects of this species.

AIM OF THE STUDY:

The goal of this study was to perform phytochemicals studies of lyophilized infusion of D. alata leaves (LI-DA), as well as obtaining ethyl acetate fraction (EAF-DA) and hydromethanolic fraction (HMF-DA), and isolated flavonoids. The antioxidant of LI-DA, EAF-DA and HMF-DA, anti-inflammatory effects of LI-DA and quercetin-3-O-β-glucoside-7-O-α-rhamnoside (DA-1) and quercetin-7-O-α-rhamnoside (DA-2) were performed while in silico tests were used for absorption, distribution, metabolism, excretion and toxicity predictions of DA-1 and DA-2.

MATERIALS AND METHODS:

LI-DA, EAF-DA and HMF-DA were evaluated in antioxidant assays (2, 2'-azino-bis (3-ethylbenzothiazoline-6-sulfonic acid - ABTS; 2,2-diphenyl-1-picrylhydrazyl - DPPH; hydrogen peroxide - H₂O₂; reducing power and oxidation of β-carotene). The DA-1 and DA-2 were isolated from EAF-DA using chromatographic methods and characterized by Nuclear Magnetic Resonance (NMR) spectrometer. The Programs ProTox 3.0 and ADMETlab 2.0 were used for the prediction studies of DA-1 and DA-2. Mice received a single dose of LI-DA (3, 30, and 100 mg/kg), DA-1 (3 mg/kg) and DA-2 (3 mg/kg) and were subjected to inflammation induced by Complete Freund's Adjuvant (CFA) and in the zymosan-induced articular inflammation model.

RESULTS:

DA-1 and DA-2 have been identified for the first time in the leaves of D. alata. LI-DA, EAF-DA and HMF-DA demonstrated a high level of antioxidant activity as measured by ABTS (IC₅₀ ≤ 5.62 μg/mL) and DPPH (IC₅₀ ≤ 11.45 μg/mL). Oral administration of LI-DA (3, 30 and 100 mg/kg), DA-1 (3 mg/kg) and DA-2 (3 mg/kg) showed significantly reduced edema, cold and mechanical allodynia in the CFA-induced inflammation model (24 hours). LI-DA (3, 30, and 100 mg/kg) and DA-1 (3 mg/kg) reduced leukocytes migration into the joint cavity, mechanical allodynia, edema and NO production in mice (24 hours) in the zymosan-induced articular inflammation model. Additionally, DA-2 (3 mg/kg) reduced leukocyte migration and LI-DA (30 mg/kg) reduced protein exudation (24 hours) in zymosan model. DA-1 and DA-2 showed good oral bioavailability and low toxicity predicted by the ProTox model.

CONCLUSION:

This is the first chemical and biological study performed of D. alata infusion and two quercetin glycoside derivatives. The results indicated promising potential for the treatment of inflammation, pain, and rheumatism, supporting the traditional use of the infusion obtained from the leaves of D. alata.

2024-07-01·BIOORGANIC & MEDICINAL CHEMISTRY

Synthesis and anticancer properties of novel dolastatin 10 analogs featuring five-membered heterocyclic rings with a linkable group at the C-terminus

Article

作者: Gellerman, Gary ; Tobi, Dror ; Patsenker, Leonid ; Panja, Akash ; Firer, Michael ; Drori, Chen ; Sharma, Vipin ; Bazylevich, Andrii ; Mitra, Pousali ; Kayet, Anirban

Dolastatin 10 (Dol-10), a natural marine-source pentapeptide, is a powerful antimitotic agent regarded as one of the most potent anticancer compounds found to date. Dol-10 however, lacks chemical conjugation capabilities, which restricts the feasibility of its application in targeted drug therapy. This limitation has spurred the prospect that chemical structure of the parent molecule might allow conjugation of the derivatives to drug carriers such as antibodies. By first employing docking studies, we designed and prepared a series of novel Dol-10 analogs with a modified C-terminus, preserving high potency of the parent compound while enhancing conjugation capability. The modifications involved the introduction of a methyleneamine functionality at position 4 of the 1,3-thiazole ring, along with the substitution of the thiazole ring with a 1,2,3-triazole moiety, furnished with methylenehydroxy, carboxy, methyleneamine, and N(Me)-methyleneamine tethering functionalities at position 4. Among the synthesized pentapeptides, DA-1 exhibited the highest potency in prostate cancer (PC-3) cells, eliciting apoptosis (IC₅₀ 0.2 ± 0.1 nm) and cell cycle arrest at the mitotic stage after at least 6 days of culture. This delayed response suggests the accumulation of cellular stress or significant physiological alterations that profoundly impact the cell cycle. We believe that these novel Dol-10 derivates represent a new and straightforward route for the development of C-terminus modified Dol-10-based microtubule inhibitors, thereby advancing targeted anticancer therapy.

2021-02-01·Cell stem cell1区 · 医学

Preclinical Efficacy and Safety of a Human Embryonic Stem Cell-Derived Midbrain Dopamine Progenitor Product, MSK-DA01

1区 · 医学

Article

作者: Riviere, Isabelle ; Irion, Stefan ; Studer, Lorenz ; Tomishima, Mark ; Hill, Ellen J ; Safford, Brent ; Bermudez, Vladimir ; Dubose, Brittany N ; Mann, Shannon ; Tabar, Viviane ; Henchcliffe, Claire ; Wang, Yongzeng ; Zabierowski, Susan ; Horn, Callie ; El Maarouf, Abderrahman ; Kriks, Sonja ; Fredrickson, Craig ; Wong, Karen ; Piao, Jinghua ; Claros, Nidia ; Rosen, Siera ; Ramnarine, Kiran ; Navare, Monalisa ; Rutishauser, Urs

Parkinson's disease is characterized by the loss of dopaminergic neurons in the substantia nigra leading to disabling deficits. Dopamine neuron grafts may provide a significant therapeutic advance over current therapies. We have generated midbrain dopamine neurons from human embryonic stem cells and manufactured large-scale cryopreserved dopamine progenitors for clinical use. After optimizing cell survival and phenotypes in short-term studies, the cell product, MSK-DA01, was subjected to an extensive set of biodistribution, toxicity, and tumorigenicity assessments in mice under GLP conditions. A large-scale efficacy study was also performed in rats with the same lot of cells intended for potential human use and demonstrated survival of the grafted cells and behavioral amelioration in 6-hydroxydopamine lesioned rats. There were no adverse effects attributable to the grafted cells, no obvious distribution outside the brain, and no cell overgrowth or tumor formation, thus paving the way for a future clinical trial.

项与 Bemdaneprocel 相关的新闻（医药）

2024-10-30

·BioSpace

10 Years of NextGen Honorees: Where Are They Now?

iStock, leolintang BioSpace has been compiling a list of the most innovative and exciting biotechs for a decade. Here we take a look back at noteworthy companies from each of those lists. 2024 marked the 10th year of BioSpace ’s NextGen list, a collection of biotechs that are on the cutting edge and poised to break barriers in the industry. As we prep next year’s list, to be released in January, we decided to take a look back at a notable honoree from each of our past lists. There were many fascinating companies that have gone on to great things; on the other hand, sometimes things don’t go as well as we predicted. We have a few not-so-great stories to tell, too. ___________________________________________ 2024 ReNAgade Therapeutics Status: Bought by Orna Therapeutics This company made our list for a pretty big reason: a massive $300 million series A in one of the toughest financing environments in recent memory. The company was launched in May 2023 to develop RNA medicines to treat diseases anywhere in the body. One of the lead investors in the round was MPM BioImpact, whose managing partner Ansbert Gadicke founded ReNAgade. Early in the biotech’s history as a stealth company, there was a bit of foreshadowing as to where the company would end up. ReNAgade struck up a partnership with Orna Therapeutics, which was also working on RNA therapeutics. The work attracted the attention of Merck in a deal worth up to $3.5 billion with development and commercialization milestones. Given the money on the line, Orna went all in on ReNAgade, buying out the biotech in May of this year—almost a year to the day after its launch. Financial details were not disclosed. See the full class of 2024 . ___________________________________________ 2023 Mariana Oncology Status: Bought by Novartis What a year 2023 turned out to be for Mariana Oncology. After raising a $175 million series B in September of that year, the company was bought out by Novartis for $1 billion , plus $750 million in milestones. Originally known as Curie Therapeutics, Mariana emerged from stealth with a $75 series A raise in December 2021 to develop new radiopharmaceuticals. The company caught our eye and made the 2023 list, but apparently Novartis was keeping an eye too. The radiopharmaceutical-hungry Big Pharma said at the time of the deal that Mariana’s technology would help it double down in the field of radioligand therapeutics. On a recent earnings call, Novartis executives expressed their excitement in the Mariana technology, saying that candidates from the buyout are speeding toward the clinic and could go after well-established antibody-drug conjugate (ADC) targets. See the full class of 2023 . ___________________________________________ 2022 Eikon Therapeutics Status: Operating Eikon has spent the two years since landing on our list racking up cash and poaching executives from CEO Rodger Perlmutter’s former employer Merck. The biotech’s raises have been huge in a really tough market: a $148 million series A in May 2021, $518 million for a January 2022 series B and most recently, $106 million in series C financing in June 2023 . At the most recent raise, Eikon also announced the acquisition of a clinical pipeline. The company now boasts oncology assets in Phase I and II testing for non-small cell lung cancer and melanoma, respectively. The rest of Eikon’s pipeline is in preclinical research or discovery. See the full class of 2022 . ___________________________________________ 2021 EQRx Status: Bought by Revolution Medicines, assets terminated EQRx emerged with a lofty goal we could all get behind: develop drugs at a lower cost. It raised a cool $200 million in a July 2020 series A, then caught the special purpose acquisition company (SPAC) wave to the Nasdaq just over a year later, adding $1.8 billion in an August 2021 IPO. The company was helmed at first by serial biotech entrepreneur Alexis Borisy. Trouble emerged shortly thereafter, with EQRx cutting lead asset sugemalimab for NSCLC after determining it had no path to market. The company shifted to two other assets, aumolertinib and lerociclib, but later dropped the former. Several rounds of layoffs made the company ever smaller before EQRx finally gave up entirely. The assets were terminated as Revolution Medicines bought out the company and its hefty $1 billion bank account. Borisy has since gone on to start a venture capital firm called Curie.Bio . See the full class of 2021 . ___________________________________________ 2020 Cerevel Therapeutics Status: Bought by AbbVie Cerevel as a company is no more, but its medicines live on at AbbVie, which bought the biotech for $8.7 billion in December 2023. The deal was part of a trend of neuroscience-focused biotechs being bought up by Big Pharma, with Cerevel moving in the same month as Bristol Myers Squibb bought Karuna Therapeutics for $14 billion. BMS was rewarded quickly with a recent FDA approval for KarXT (now Cobenfy) for schizophrenia, but AbbVie too has had success since sealing the deal. Cerevel’s investigational Parkinson’s disease therapy tavapadon improved control of motor symptoms when used with common treatment levodopa in a Phase III trial that read out in April. The data would support a regulatory filing for the therapy, executives said at the time. Two additional late-stage readouts for the therapy are due later this year. But the real star of the deal was emraclidine, which is in Phase III studies for schizophrenia. Cerevel also brought a major depression treatment to the table. “These assets clearly will be great additions to our neuroscience franchise,” said AbbVie’s CEO Rob Michael on the company’s second quarter earnings call. See the full class of 2020 . ___________________________________________ 2019 Genevant Status: Operating One of Roivant’s offshoots, Genevant is still operating today, working on developing nucleic acid delivery technology. Just last week, Genevant signed a deal with CRISPR gene editing company Editas Medicines to develop in vivo gene editing medicines for two undisclosed targets. Genevant has also recently signed deals with Novo Nordisk, Tome Biosciences, Gritstone bio and Repair Biotechnologies. But perhaps the most compelling story from Genevant since we named them to our NextGen list has been their litigation with famed biotech Moderna over the lipid nanoparticle delivery system used for its approved COVID-19 vaccine. Genevant and Arbutus Biopharma filed several patent infringement claims against Moderna arguing that the vaccine developer used their technology without license or proper compensation. A judge sided with Arbutus and Genevant in April, with a trial set for next year. See the full class of 2019 . ___________________________________________ 2018 BlueRock Therapeutics Status: Bought by Bayer Bayer bought cell therapy biotech BlueRock Therapeutics in August 2019 for $1 billion, but rather than absorbing the company into itself, the German pharma kept the name and unit operating. This unusual but refreshing strategy means that the company essentially lives on. In June 2023, BlueRock reported that Parkinson’s disease stem cell therapy bemdaneprocel was well tolerated with no significant side effects in a Phase I trial with 12 patients. It was the first cell therapy to show positive results in human testing, Bayer said at the time. A September update showed similar safety results. The therapy has since received a regenerative medicine advanced therapy tag from the FDA. Elsewhere in the pipeline, BlueRock’s investigational iPSC-derived cell therapy OpCT-001 for certain retinal disorders was cleared for human testing in September. The therapy was licensed from FUJIFILM Cellular Dynamics and Opsis Therapeutics. See the full class of 2018 . ___________________________________________ 2017 Decibel Therapeutics Status: Bought by Regeneron Regeneron made a splash at this year’s American Society of Gene and Cell Therapy meeting in Baltimore when it showcased results of a Phase I/II trial of hearing loss therapy DB-OTO. The therapy restored hearing in two children who had a genetic form of hearing loss. While Regeneron’s branding was behind the conference presentation, the gene therapy came from Decibel Therapeutics. Regeneron bought the biotech for $109 million in August 2023, at which point the two companies had already been working together for six years. The biotech was integrated into Regeneron’s genetic medicines portfolio. See the full class of 2017 . ___________________________________________ 2016 CRISPR Therapeutics Status: Operating CRISPR Therapeutics made history last year as the first company, along with partner Vertex Pharmaceuticals, to gain an FDA approval for a CRISPR-based gene edited therapy. The companies were granted an FDA greenlight for Casgevy in sickle cell disease. The same day, bluebird bio got a similar nod for a competing gene therapy, though not one that involves CRISPR technology. The FDA approval was a triumph of modern biotechnology. But the roll out has been slow given the nature of gene therapy treatment, and only recently have patients begun receiving infusions of the medicine. Casgevy is now also approved for beta thalassemia, another blood disorder. The company has turned to the rest of its pipeline, including a CD19-directed CAR T cell program called CTX112, which is being tested in both cancer and autoimmune disorders. CTX131 is in development for hematological malignancies as well. CEO Sam Kulkarni has expressed confidence in CRISPR’s leading position in gene editing and pledged to be on top of emerging technologies. “One of the most important considerations for us is that we don’t get disrupted,” he said at the Goldman Sachs healthcare conference in June. “We are the leaders in the gene editing space and we have been, but we want to make sure that we’re looking at every improvement that’s coming out there.” See the full class of 2016 . ___________________________________________ 2015 Spark Therapeutics Status: Bought by Roche Like BlueRock, Spark Therapeutics was folded into a Big Pharma but kept its identity. Spark earned a spot on our very first NextGen list in 2015 thanks to its active clinical hemophilia program and fundraises totaling $122.8 million. The company caught the eye of Roche, which swooped in with a $4.8 billion bid in February 2019. The deal eventually turned into a bit of a boondoggle, with regulatory delays slowing things down until it finally closed that December. At issue was Roche’s hemophilia Hemlibra. Regulators worried that Spark’s hemophilia program and Hemlibra together could stifle competition and create a monopoly. Spark brought with it the approved eye disease gene therapy Luxturna. With the deal closed, Spark has charged forward under the Roche banner. The unit is developing a 500,000-square foot Gene Therapy Innovation Center in Philadelphia as its flagship manufacturing space. See the full class of 2015 .

临床1期并购临床3期上市批准

2024-10-15

·MedCity News

On Heels of Positive Parkinson’s Gene Therapy Data, MeiraGTx Looks Ahead to Pivotal Test - MedCity News

A MeiraGTx Holdings gene therapy for Parkinson’s disease has preliminary data from a small clinical trial showing the one-time treatment showed both safety and efficacy, results that lay the groundwork for discussions with regulators about a Phase 3 study. The MeiraGTx gene therapy, AAV-GAD, had previously posted Phase 1 and Phase 2 results showing it was well tolerated with no significant adverse effects. The results announced Tuesday are from a Phase 1 bridging study designed to evaluate doses of AAV-GAD manufactured by London-based MeiraGTx at its own facilities using its commercial platform process. Parkinson’s patients lack sufficient amounts of a neurotransmitter called dopamine. Standard treatment includes the drug levodopa, a compound that a brain enzyme converts into dopamine. But dopamine replacement therapies lose efficacy over time. The motor symptoms of Parkinson’s can also be treated with deep brain stimulation, but that’s an invasive procedure. sponsored content Upcoming Webinar: Battle of the Budget: Employers vs. Rising Healthcare Costs Gain valuable knowledge and practical insights on how employers are addressing an increasingly complex healthcare budget landscape. By Personify Health MeiraGTx’s gene therapy is designed to spark local production of a neurotransmitter called GABA, which is intended to reprogram dysfunctional brain circuits and restore more normal activity to cells, alleviating motor symptoms. The 26-week bridging study enrolled Parkinson’s patients who had a history of responsiveness to levodopa treatment for at least 12 months. A total of 14 participants were randomly assigned to receive a high dose, a low dose, or a sham treatment. According to MeiraGTx, AAV-GAD met the main goal of safety and tolerability with no serious adverse events (SAEs) reported. Efficacy, an exploratory endpoint, was evaluated by measuring motor examination scores. The company said at week 26, results show a statistically significant 18-point average improvement from baseline according to a rating scale used to measure the severity and progression of Parkinson’s. By comparison, no significant change was observed in the low-dose group or the sham cohort. “With material made using our proprietary production process at commercial scale, we have demonstrated that AAV-GAD is safe at all doses studied, including a higher dose than previously tested,” MeiraGTx President and CEO Alexandria Forbes said in a prepared statement. “We have now treated a total of 58 patients in this development program in three independent multicenter clinical studies and have seen no SAEs related to AAV-GAD treatment.” Participants who completed the bridging study have the option of enrolling in a long-term follow-up study, which will monitor them for five years. Meanwhile, MeiraGTx said it plans to discuss the results with regulators in the U.S., Europe, and Japan as it looks ahead to pivotal testing of the gene therapy. presented by Sponsored Post The Promise of Value-Based Care and MedTech Innovation Monica Vajani, Executive Director for mHUB’s MedTech Accelerator, discusses how mHUB is helping innovators transition healthcare towards value-based care. By Monica Vajani - mHUB MeiraGTx isn’t the only company working to develop a one-time treatment for Parkinson’s. Bayer’s bemdaneprocel is made by engineering donor cells, turning them into dopamine-producing neurons. These cells are surgically transplanted into a patient’s brain where they are intended to replace a patient’s lost dopamine production. Last year, Bayer reported positive safety and efficacy data from bemdaneprocel’s Phase 1 test. Last month, Bayer reported these encouraging trends continued measured at 24 months. Aspen Neuroscience is also developing a Parkinson’s cell therapy. But unlike Bayer’s approach, Aspen’s ANPD001 is made from stem cells sourced from a patient’s own skin, offering the potential to avoid immunosuppressive drugs needed to prevent the body from rejecting the transplanted cells. Last month, Aspen announced it had completed dosing of the first cohort of a Phase 1/2a test of this cell therapy. Image: Dr_Microbe, Getty Images

临床结果细胞疗法临床1期基因疗法临床3期

2024-10-09

·PRNewswire

Cell and Gene Therapy in Parkinson's Disease Clinical Trial Pipeline Analysis Demonstrates 18+ Key Companies at the Horizon Expected to Transform the Treatment Paradigm, Assesses DelveInsight

The market for cell and gene therapy in Parkinson's disease is driven by the critical unmet medical need for disease-modifying treatments, advancements in clinical trials showing promising results, and regulatory support for innovative therapies. Additionally, the economic burden of managing PD and the rise in patient advocacy for cutting-edge solutions are further accelerating investment and development in this area. Together, these factors position cell and gene therapies as transformative in addressing the current treatment gaps in Parkinson's disease. LAS VEGAS, Oct. 9, 2024 /PRNewswire/ -- DelveInsight's ' Cell and Gene Therapy in Parkinson's Disease Pipeline Insight – 2024 ' report provides comprehensive global coverage of pipeline cell and gene therapy in Parkinson's disease in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space and future growth potential of the cell and gene therapy in Parkinson's disease pipeline domain. Key Takeaways from the Cell and Gene Therapy in Parkinson's Disease Pipeline Report DelveInsight's cell and gene therapy in Parkinson's disease pipeline report depicts a robust space with 18+ active players working to develop 20+ cell and gene therapies for Parkinson's disease treatment. Key cell and gene therapy in Parkinson's disease companies such as Sumitomo Dainippon Pharma, BlueRock Therapeutics, Aspen Neuroscience, Prevail Therapeutics, S.Biomedics, MeiraGTx, Asklepios BioPharmaceutical, iRegene Therapeutics, Kenai Therapeutics, Tetraneuron, and others are evaluating new cell and gene therapy in Parkinson's disease to improve the treatment landscape. Promising pipeline cell and gene therapy in Parkinson's disease in various stages of development include CT1 DAP001/DSP 1083, BRT-DA01, ANPD 001, LY3884961, A9-DPC, AAV-GAD, AB-1005, NouvNeu 001, RNDP 003, TET AAV E2F4DN, and others. In July 2024, S.BIOMEDICS announced positive one-year assessment data from the first 3 participants (first low dose cohort) of Phase I/IIa clinical trial for Parkinson's disease with TED-A9, hESC (human embryonic stem cell)-derived midbrain dopaminergic progenitors. In July 2024, Bayer AG and Asklepios BioPharmaceutical announced that the United States (U.S.) Food and Drug Administration (FDA) had granted Fast Track Designation for AB-1005, which is being developed for moderate Parkinson's disease. In June 2024, The U.S. Food and Drug Administration (FDA) approved the IND application for NouvNeu001, a cell therapy product from iRegene therapeutics targeting Parkinson's Disease. Previously, the "Combined Phase I/II Clinical Study" of NouvNeu001 had already received approval from China NMPA in August 2023 and demonstrated positive safety and efficacy data in its Phase I trials. In May 2024, Bayer AG and BlueRock Therapeutics announced that BlueRock's investigational cell therapy bemdaneprocel for the treatment of Parkinson's disease had been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). In April 2024, Aspen Neuroscience, Inc. announced that the first patient had been dosed in the ASPIRO trial, a Phase I/IIa open label clinical trial to assess safety and tolerability of ANPD001, an autologous, dopaminergic neuron cell replacement therapy for participants with moderate to severe Parkinson's disease (PD). In March 2024, Bayer AG and BlueRock Therapeutics announced details of 18-month data from the phase I clinical trial for bemdaneprocel (BRT-DA01), an investigational allogeneic stem cell derived cell therapy for treating Parkinson's disease. In January 2024, Bayer AG and Asklepios BioPharmaceutical announced the completion of the 18-month data collection in the Phase Ib clinical trial for AB-1005 (AAV2-GDNF), an investigational gene therapy for treating patients with Parkinson's disease (PD).1, Request a sample and discover the recent advances in cell and gene therapy in Parkinson's disease drug treatment @ Cell and Gene Therapy in Parkinson's Disease Pipeline Report The cell and gene therapy in Parkinson's disease pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage cell and gene therapy in Parkinson's disease, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the cell and gene therapy in Parkinson's disease clinical trial landscape. Cell and Gene Therapy in Parkinson's Disease Overview Cell and gene therapy holds significant promise for treating Parkinson's disease, a neurodegenerative disorder characterized by the progressive loss of dopaminergic neurons in the brain. Cell therapy focuses on the transplantation of stem cells or progenitor cells into the brain to replace the lost neurons or support their survival. These transplanted cells can potentially differentiate into dopaminergic neurons, helping restore the dopamine levels that are crucial for motor control. Recent advances in induced pluripotent stem cells (iPSCs) have enabled the development of patient-specific neurons, which can minimize immune rejection and offer personalized treatment options. Early clinical trials have shown potential, though challenges remain in ensuring long-term survival and functionality of the transplanted cells. Gene therapy, on the other hand, aims to modify the genetic makeup of neurons to slow down or reverse the disease process. Techniques like viral vectors are used to deliver genes that can boost dopamine production, protect neurons from degeneration, or reduce the buildup of harmful proteins like alpha-synuclein, which is implicated in Parkinson's disease pathology. One notable example is the delivery of the GDNF (glial cell line-derived neurotrophic factor) gene, which has been shown to support the survival of dopaminergic neurons. Although gene therapy for Parkinson's disease is still in its experimental stages, it holds potential as a long-lasting and less invasive approach compared to traditional treatments like deep brain stimulation or dopamine replacement therapy. With ongoing advancements, both cell and gene therapies could revolutionize how Parkinson's disease is managed in the future. Find out more about cell and gene therapy in Parkinson's disease @ New Cell and Gene Therapies in Parkinson's Disease A snapshot of the pipeline Cell and Gene Therapy in Parkinson's Disease mentioned in the report: Learn more about the emerging cell and gene therapy in Parkinson's disease @ Cell and Gene Therapy in Parkinson's Disease Clinical Trials Cell and Gene Therapy in Parkinson's Disease Therapeutics Assessment The cell and gene therapy in Parkinson's disease pipeline report proffers an integral view of emerging cell and gene therapy in Parkinson's disease segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Cell and Gene Therapy in Parkinson's Disease Pipeline Report Coverage: Global Cell and Gene Therapy in Parkinson's Disease Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Cell and Gene Therapy in Parkinson's Disease Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Cell and Gene Therapy in Parkinson's Disease Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Cell and Gene Therapy in Parkinson's Disease Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Key Cell and Gene Therapy in Parkinson's Disease Companies: Sumitomo Dainippon Pharma, BlueRock Therapeutics, Aspen Neuroscience, Prevail Therapeutics, S.Biomedics, MeiraGTx, Asklepios BioPharmaceutical, iRegene Therapeutics, Kenai Therapeutics, Tetraneuron, and others. Key Cell and Gene Therapy in Parkinson's Disease Pipeline Therapies: CT1 DAP001/DSP 1083, BRT-DA01, ANPD 001, LY3884961, A9-DPC, AAV-GAD, AB-1005, NouvNeu 001, RNDP 003, TET AAV E2F4DN, and others. Dive deep into rich insights for new cell and gene therapy for Parkinson's disease treatment; visit @ Cell and Gene Therapy for Parkinson's Disease Treatment Table of Contents For further information on the cell and gene therapy in Parkinson's disease pipeline therapeutics, reach out @ Cell and Gene Therapy in Parkinson's Disease Drug Treatment Related Reports Cell and Gene Therapy in Parkinson's Disease Market Cell and Gene Therapy in Parkinson's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key cell and gene therapy in Parkinson's disease companies, including MeiraGTx, Hope Biosciences, Sumitomo Pharma, Prevail Therapeutics, BlueRock Therapeutics, Voyager Therapeutics, among others. Parkinson's Disease Pipeline Parkinson's Disease Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Parkinson's disease companies, including Cerevel Therapeutics, Inhibikase Therapeutics, Neuraly, Peptron, Biogen, Roche, Brain Neurotherapy Bio, Inc., Modag, Annovis Bio Inc., BioVie Inc., United Neuroscience Ltd., Luye Pharma Group, AbbVie, UCB Biopharma SRL, InnoMedica Schweiz AG, Integrative Research Laboratories AB, H. Lundbeck A/S, Shanghai WD Pharmaceutical Co., Ltd., Cerevance Beta, Inc., Nobilis Therapeutics Inc., BlueRock Therapeutics, Taiwan Mitochondrion Applied Technology Co., Ltd., among others. Psychosis in Parkinson's and Alzheimer's Disease Market Psychosis in Parkinson's and Alzheimer's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key psychosis in Parkinson's and Alzheimer's disease companies, including Sunovion Pharmaceuticals, Karuna Therapeutics, Vanda Pharmaceuticals, Suven Life Sciences, Enterin, Intra-Cellular Therapies, Merck Sharp & Dohme, among others. Parkinson's Disease-Related Dementia Market Parkinson's Disease-Related Dementia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Parkinson's disease-related dementia companies, including AbbVie, UCB Biopharma SRL, InnoMedica Schweiz AG, Integrative Research Laboratories AB, H. Lundbeck A/S, Shanghai WD Pharmaceutical Co., Ltd., Cerevance Beta, Inc., among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . 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临床1期快速通道细胞疗法

100 项与 Bemdaneprocel 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
帕金森病	临床1期	美国	BlueRock Therapeutics LP	2021-05-03
帕金森病	临床1期	美国	Memorial Sloan Kettering Cancer Center	2021-05-03
帕金森病	临床1期	加拿大	Memorial Sloan Kettering Cancer Center	2021-05-03
帕金森病	临床1期	加拿大	BlueRock Therapeutics LP	2021-05-03

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临床结果

适应症

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ASGCT2024	临床1期	帕金森病	12	Bemdaneprocel low dose	製廠鑰壓艱觸齋觸鑰鑰(淵膚鹹鏇觸壓蓋鹹繭齋) = 衊積鹹觸憲網醖積醖齋遞憲醖鏇觸鹽簾簾廠鏇 (鏇淵廠選糧構顧獵顧製 ) 更多	积极	2024-05-07
				Bemdaneprocel high dose	製廠鑰壓艱觸齋觸鑰鑰(淵膚鹹鏇觸壓蓋鹹繭齋) = 膚糧窪顧淵鏇製顧壓廠遞憲醖鏇觸鹽簾簾廠鏇 (鏇淵廠選糧構顧獵顧製 ) 更多
NCT04802733 (ASGCT2024) 人工标引	临床1期	帕金森病	12	Bemdaneprocel	廠糧顧鹽鹽鏇窪襯艱繭(齋窪壓衊蓋窪範築廠製) = 積鏇衊獵觸襯鏇壓夢簾醖觸夢繭願蓋網膚鑰艱 (壓夢顧繭憲襯廠顧餘鹹 ) 更多	积极	2024-04-23
AAN2024	临床1期	帕金森病	12	Bemdaneprocel low-dose	鑰廠糧觸鑰網網繭襯選(廠構築願衊膚網鏇鬱築) = 壓範蓋獵鹹膚衊齋製夢衊獵蓋餘衊顧繭積齋夢 (鹽齋襯鬱願鏇鏇膚選鏇, -0.4 ~ 1.0)	积极	2024-04-09
				Bemdaneprocel high-dose	鑰廠糧觸鑰網網繭襯選(廠構築願衊膚網鏇鬱築) = 簾衊蓋鹽醖鏇製窪壓遞衊獵蓋餘衊顧繭積齋夢 (鹽齋襯鬱願鏇鏇膚選鏇, -0.4 ~ 2.2)
NCT04802733 (Corporate Publications) 人工标引	临床1期	帕金森病	12	Bemdaneprocel (BRT-DA01) 0.9 million cells per putamen	選遞壓築顧襯糧齋獵鹽(獵艱製壓鏇襯鏇夢膚顧) = 網醖餘餘夢鏇蓋襯選艱餘鏇築窪淵窪選繭膚廠 (顧構鹽範顧簾遞構餘蓋 ) 更多	积极	2024-03-06
				Bemdaneprocel (BRT-DA01) 2.7 million cells per putamen	選遞壓築顧襯糧齋獵鹽(獵艱製壓鏇襯鏇夢膚顧) = 遞淵餘艱網鏇窪遞選構餘鏇築窪淵窪選繭膚廠 (顧構鹽範顧簾遞構餘蓋 ) 更多
Phase 1 Study of Bemdaneprocel (ANA2023)	临床1期	帕金森病	12	Bemdaneprocel low dose	網觸觸壓願衊繭壓構憲(糧壓觸淵遞衊餘衊淵壓) = 齋壓選繭顧糧壓鏇衊窪鹹築遞構醖顧鹽鏇餘齋 (網壓積簾構選簾餘蓋願, 1.4 ~ 6.2) 更多	积极	2023-09-01
				Bemdaneprocel high dose	網觸觸壓願衊繭壓構憲(糧壓觸淵遞衊餘衊淵壓) = 壓蓋糧製鹹襯網齋壓積鹹築遞構醖顧鹽鏇餘齋 (網壓積簾構選簾餘蓋願, 1.4 ~ 6.2) 更多
NCT04802733 (PRNewswire) 人工标引	临床1期	帕金森病	12	BRT-DA01 (high dose cohort)	糧艱膚膚膚餘壓淵觸糧(衊鏇獵膚醖積淵醖壓積) = no serious adverse events (SAEs) reported related to bemdaneprocel through one year 襯膚遞遞壓簾獵糧製糧 (繭餘遞襯積繭憲糧網艱 )	积极	2023-08-28
				BRT-DA01 (low dose cohort)
MDS2023	临床1期	帕金森病	12	Bemdaneprocel low dose	選壓遞鑰醖壓膚膚鑰構(壓鹹夢壓鏇選憲願廠糧) = 襯襯願選鹽憲廠簾繭鹹範遞繭獵鹽衊選膚夢鹹 (壓壓築廠鏇鏇鹽遞衊淵 )	-	2023-08-27