Retinal pigment epithelium

Verastem Oncology Announces Positive Initial Interim Safety and Efficacy Results from RAMP 205 Trial Evaluating Avutometinib Plus Defactinib in Combination with Gemcitabine and Nab-paclitaxel in First-Line Metastatic Pancreatic Cancer​

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木曜日, 5月 23, 2024
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Therapy, Sepsis, Patient, NAB, Retinal pigment epithelium, Bile, RAMP, Toxicity, ECOG, Febrile neutropenia, Verastem Oncology, Septic shock, BID, Clinical trial, Male, EDT, Avutometinib, Disease, Safety, Skin infection, Cancer, Pancreatic cancer, MMSc, Metastasis, ASCO, Anemia, Șaeș, Society, Eastern Cooperative Oncology Group, Mutation, Pulmonary embolism, Pneumoperitoneum, Webcast, BIW, Vomiting, DLT, Medicine, Annual general meeting, Phosphorus

Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today announced the initial interim safety and efficacy results from the ongoing RAMP 205 Phase 1/2 clinical trial evaluating avutometinib plus defactinib in combination with gemcitabine and Nab-paclitaxel in the first-line in patients with metastatic pancreatic cancer.

Key Points: 
  • Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today announced the initial interim safety and efficacy results from the ongoing RAMP 205 Phase 1/2 clinical trial evaluating avutometinib plus defactinib in combination with gemcitabine and Nab-paclitaxel in the first-line in patients with metastatic pancreatic cancer.
  • We continue to progress the study evaluating other dose and schedule regimens to determine the recommended Phase 2 dose in the trial,” said John Hayslip, M.D., chief medical officer of Verastem Oncology.
  • “We look forward to Verastem presenting their initial data from the Phase 1b/2a trial of avutometinib and defactinib in combination with standard care gemcitabine and Nab-paclitaxel in previously untreated metastatic pancreatic cancer at ASCO.
  • Verastem will hold an investor conference call and webcast on Friday, May 24 at 8:00 am EDT, to discuss these data.

Vesigen Highlights Key in vivo and Targeted Tropism Data Advancements for Non-Viral Delivery Platform at 2024 ASGCT and ARVO Annual Meetings

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月曜日, 5月 13, 2024
Research, Neurology, Genetics, Biotechnology, Health, Pharmaceutical, General Health, Other Science, Science, Cell, GMP, IRF5, Retinal pigment epithelium, FA, Friedreich's ataxia, NHP, Stargardt disease, Therapy, ABCA4, Safety, RPE, Engineering, Administration, Stargardt, Gene, Tissue, Neuron, NLRP3, RNA, Society, Retina, Inflammation, ALI, ARVO, GAA, Gene editing, Annual general meeting, Vaccine

ARVO was held from May 5-9 in Seattle, Washington, while ASGCT was held from May 7-11, 2024 in Baltimore, Maryland.

Key Points: 
  • ARVO was held from May 5-9 in Seattle, Washington, while ASGCT was held from May 7-11, 2024 in Baltimore, Maryland.
  • “We’ve made substantial strides with our ARMMs platform since last year’s ASGCT Annual Meeting.
  • ARMMs were decorated with engagers specific to proprioceptive neurons, potentially enabling in vivo delivery to disease-affected cell types in FA.
  • Two data presentations (ASGCT #1246 and #1746) highlighted continued development and optimization of scalable approaches to produce and characterize ARMMs.

Annexon Presents New Neuroprotection Data Showing ANX007 Protects Vision and Vision-Associated Structures in Geographic Atrophy at ARVO 2024 Annual Meeting

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火曜日, 5月 7, 2024
Brain, ARCHER, Complement component 1q, CNV, Research, Microglia, Incidence, Patient, Eye, Pivotal, Vasculitis, Paratriathlon, Retinal pigment epithelium, Geographic atrophy, RPE, Atrophy, Annual general meeting, Synapse, ARVO, European Medicines Agency, Overalls, Visual acuity, Medical imaging

BRISBANE, Calif., May 07, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a biopharmaceutical company advancing a late-stage clinical platform of novel therapies for people living with devastating classical complement-mediated neuroinflammatory diseases of the body, brain, and eye, today presented new analyses of ANX007 from the Phase 2 ARCHER trial in geographic atrophy (GA), and new preclinical data on the role of C1q in the pathogenic elimination of photoreceptor synapses and their protection with C1q blockade in GA. ANX007 is a first-in-class, non-pegylated antigen-binding fragment (Fab) designed to block C1q and activation of the classical complement cascade locally in the eye with an intravitreal formulation, and is the first therapeutic candidate for the treatment of GA to receive Priority Medicine (PRIME) designation by the European Medicines Agency. The data were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting.

Key Points: 
  • The data were presented at the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting.
  • “We are pleased to present additional clinical data from the ARCHER trial that are the first to show preservation of both vision and relevant anatomical structures following ANX007 treatment,” said Douglas Love, president and chief executive officer of Annexon.
  • “Importantly, the statistically significant preservation of photoreceptor anatomy measured by ellipsoid zone change highlights protection of key retinal structures associated with vision.
  • These two registrational trials are designed to confirm the Phase 2 ARCHER findings of protection against vision loss and underscore the unique neuroprotective mechanism of action of ANX007 and its competitive differentiation in visual function.

PulseSight Therapeutics Launches to Advance Non-viral Gene Therapies with Disruptive Minimally-Invasive Delivery Technology for Severe Retinal Diseases

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水曜日, 2月 28, 2024
Doctor of Philosophy, Decorin, Drug development, Sale, Iris (anatomy), Transferrin, Ciliary muscle, Geographic atrophy, Ranibizumab, Iron, Retinitis pigmentosa, Retina, CNV, Paratriathlon, Eye, Brolucizumab, VEGF, Therapy, AMD, Independent, DNA, Retinal pigment epithelium, KIP, Doctor of Pharmacy, Safety, Novartis, Oxidative stress, CSO, Conditional sentence, RPE, EMA, MD, FDA, SAS, Choroidal neovascularization, Cell, Human, Patient, Glaucoma, Gene expression

Unique proprietary non-viral gene therapy platform with minimally invasive delivery technology providing long lasting gene expression and favorable distribution in the retina.

Key Points: 
  • Unique proprietary non-viral gene therapy platform with minimally invasive delivery technology providing long lasting gene expression and favorable distribution in the retina.
  • A substantially de-risked platform and two first-in-class gene therapies heading to the clinic, having the potential to become future blockbusters.
  • PARIS, Feb. 28, 2024 (GLOBE NEWSWIRE) -- PulseSight Therapeutics SAS, an ophthalmology biotech company developing disruptive non-viral gene therapies with minimally-invasive delivery technology, launches today with seed finance from Pureos Bioventures and ND Capital.
  • PulseSight is currently raising a Series A financing round to advance its programs into clinical proof-of-concept.

RxCell Announces $4,600,000 Investment from DMR Global Venture and Regenosis, Plans for Second Raise of $15,000,000 for IND Testing in Q4 2023

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火曜日, 12月 5, 2023
Infectious Diseases, Finance, Clinical Trials, Professional Services, Stem Cells, Biotechnology, Health, Pharmaceutical, General Health, RPE, SERI, National University of Singapore, Notifiable disease, IND, Patient, USD, Investigational New Drug, Cell death, NUS, AMD, RPC, Retinal pigment epithelium, Cell, Name, National university, Pharmaceutical industry, Medical device

RxCell, a pioneering biotech company focusing on cellular therapeutics for age-related diseases, today proudly announces a $4,600,000 investment commitment from DMR Global Venture LLC and Regenosis.

Key Points: 
  • RxCell, a pioneering biotech company focusing on cellular therapeutics for age-related diseases, today proudly announces a $4,600,000 investment commitment from DMR Global Venture LLC and Regenosis.
  • Furthermore, RxCell is gearing up for a substantial second capital raise of $15,000,000 in Q4 2023.
  • The global Age-related Macular Degeneration (AMD) Market, an integral segment RxCell operates in, had a valuation of USD 7.47 billion in 2021.
  • Professor Xianmin Zeng, CEO of RxCell, remarked, "We're thrilled to align with DMR Global Venture and Regenosis.

Vesigen Highlights Non-Human Primate Biodistribution of Non-Viral Technology and Functional Delivery of Genome Editors In Vivo at the Cracking the Code: The Dawn of Nucleic Acid Medicines Meeting

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木曜日, 10月 19, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Gene editing, Retinal pigment epithelium, Mouse, Dawn, RNA, ARMM, Histopathology, ARMM Regional Legislative Assembly, RPE, Disease, Cell death, Cytotoxicity, Therapy, Kupffer cell, Retina, Vaccine

Specifically, the Company presented new data demonstrating retinal biodistribution of engineered ARMMs across multiple species – including non-human primates – and data demonstrating successful in vivo functional delivery of a Cas9/guide RNA gene editing complex.

Key Points: 
  • Specifically, the Company presented new data demonstrating retinal biodistribution of engineered ARMMs across multiple species – including non-human primates – and data demonstrating successful in vivo functional delivery of a Cas9/guide RNA gene editing complex.
  • The data were presented at the Cracking the Code: The Dawn of Nucleic Acid Medicines meeting, taking place October 17-19, 2023, in Worcester, MA.
  • “We have shown that our ARMM technology can successfully deliver functionally active gene editing complexes to Kupffer cells and ameliorate disease in an acute liver injury model.
  • Additional data in mice, minipigs, and non-human primates demonstrated the efficient delivery of subretinally administered ARMMs to the RPE and photoreceptors, cell types associated with multiple retinal diseases.

Ocumension Therapeutics Announces 2023 Interim Results

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木曜日, 8月 24, 2023
Retina, Tissue, Glaucoma, Chorioretinitis, Betaxolol, Marketing, Culture, Uveitis, COVID-19, NDA, Visual impairment, Quality of life, Vitreous body, Research, Paratriathlon, Inflammation, Philosophy, Hospital, Latanoprost/timolol, Patient, Optic nerve, CDE, Growth, Near-sightedness, Emedastine, Therapy, Safety, III, NMPA, Cryopyrin-associated periodic syndrome, Retinal pigment epithelium, Disease, Pharmaceutical industry, Fine chemical, Period, PRC

HONG KONG, Aug 24, 2023 - (ACN Newswire) - Ocumension Therapeutics ("Ocumension" or the "Company", together with its subsidiaries, collectively the "Group", stock code: 1477), a leading China ophthalmic pharmaceutical platform company, announced its interim results for the six months ended 30 June 2023 ("the Period") today.

Key Points: 
  • HONG KONG, Aug 24, 2023 - (ACN Newswire) - Ocumension Therapeutics ("Ocumension" or the "Company", together with its subsidiaries, collectively the "Group", stock code: 1477), a leading China ophthalmic pharmaceutical platform company, announced its interim results for the six months ended 30 June 2023 ("the Period") today.
  • During the Period, OcuMension maintained its focus on the pilot scale production and validation batch production for its products, such as Emadine, at its Suzhou manufacture site.
  • The Company expects to complete the enrollment of subjects for phase II clinical trials in the second half of 2023.
  • Mr. Ye LIU, CEO and Executive Director of Ocumension, said: "Over the past three years, we have made many remarkable achievements.

Clearside Biomedical Announces Second Quarter 2023 Financial Results and Provides Corporate Update

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月曜日, 8月 14, 2023
Pharming, Safety, New Drug Application, General partnership, TKI, Experiment, Patient, Research and development, R, Axitinib, SCS, SAB, ARVO, Retinal pigment epithelium, Therapy, ISO, Experimental & Molecular Medicine, Research, Genome, Choi, International Organization for Standardization, Conference call, Partner, The Association, Conference, ASRS, Retina, Eye, Administration, Chairperson, Certification, Webcast, International Organization (journal), OASIS, Association for Research in Vision and Ophthalmology, AMD, NDA, Needle, ET, Physician, Annual general meeting, Visual acuity, Society, Pharmaceutical industry, Medical device, Video game, Clearside, Ophthalmology, MD

ALPHARETTA, Ga., Aug. 14, 2023 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), today reported financial results for the second quarter ended June 30, 2023 and provided a corporate update.

Key Points: 
  • ET -
    ALPHARETTA, Ga., Aug. 14, 2023 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), today reported financial results for the second quarter ended June 30, 2023 and provided a corporate update.
  • License Revenue: License and other revenue for the second quarter of 2023 was $1.0 million, compared to $0.4 million for the second quarter of 2022.
  • Other Income: Other income for the second quarter of 2023 was $0.5 million, compared to $24,000 for the second quarter of 2022.
  • Other Expense: Non-cash interest expense for the second quarter of 2023 was $2.3 million, compared to $0 in the second quarter of 2022.

Apellis Announces Seven Abstracts Accepted for Oral Presentation at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting

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月曜日, 7月 10, 2023
Abstract, Caregiver, APLS, AMD, GALE, Geographic atrophy, GA, Angiography, OAKS, Retinal pigment epithelium, MD, Digital, FDA, Society, MOSAIC, Clinical, ASRS, Pharmaceutical industry, Communications satellite, Patient

WALTHAM, Mass., July 10, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced that seven abstracts will be presented as oral presentations at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting to be held July 28 to August 1 in Seattle.

Key Points: 
  • WALTHAM, Mass., July 10, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced that seven abstracts will be presented as oral presentations at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting to be held July 28 to August 1 in Seattle.
  • The upcoming data further reinforces the efficacy and safety profile of SYFOVRE® (pegcetacoplan injection), the first and only treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
  • “The breadth of SYFOVRE data at ASRS is a testament to the excitement surrounding the first and only FDA approved treatment for GA, which provides flexible dosing and slows GA lesion growth with increasing treatment effects over time,” said Caroline Baumal, MD, chief medical officer, Apellis.
  • “Among our seven oral presentations this year, we are thrilled to present data for the first time from the GALE long-term extension study of SYFOVRE.”
    Long-Term Efficacy of Pegcetacoplan in Patients with Geographic Atrophy – Nathan Steinle, MD – Dry AMD Symposium 2 – Sunday, July 30 – 9:06-9:12 a.m. PT
    Effect of Pegcetacoplan on Photoreceptor and Retinal Pigment Epithelium Integrity in Geographic Atrophy in the Phase 3 Trials and GALE Extension Study – Ursula Schmidt-Erfurth, MD – Dry AMD Symposium 2 – Sunday, July 30 – 9:12-9:18 a.m. PT
    Retinal Tissue Preservation with Intravitreal Pegcetacoplan in Patients with Geographic Atrophy – Sunir Garg, MD – Dry AMD Symposium 2 – Sunday, July 30 – 9:18-9:22 a.m. PT
    Pegcetacoplan vs Avacincaptad Pegol in Patients with Geographic Atrophy: An Anchored Matching-Adjusted Indirect Comparison of the Phase 3 Trials – Paul Hahn, MD – Dry AMD Symposium 2 – Sunday, July 30 – 9:42-9:46 a.m. PT
    The MOSAIC Study: A Clinical and Humanistic Burden of Illness Study of Patients with Geographic Atrophy and their Caregivers in the United States and Canada – Sophie J. Bakri, MD – Dry AMD Symposium 1 – Saturday, July 29 – 9:15-9:19 a.m. PT
    Utility of Outer Retinal Tubulation in Predicting Lesion Growth in Both Subfoveal and Nonsubfoveal Geographic Atrophy – Srinivas Kondapalli, MD – Imaging, Digital, Angiography Symposium – Saturday, July 29 – 2:57-3:01 p.m. PT
    Exudative Age-Related Macular Degeneration Events from the OAKS and DERBY Clinical Trials of Pegcetacoplan in Geographic Atrophy – Roger Goldberg, MD – Wet AMD Symposium 3 – Monday, July 31 – 1:05-1:09 p.m. PT

Adverum Biotechnologies Introduces an Intravitreal Gene Therapy Program for Geographic Atrophy and Presents Data on Its Ocular Gene Therapy Platform

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木曜日, 5月 18, 2023
Injection, CFI, Vance DeGeneres, Gene, MOI, AMD, CMC, Optogenetics, Research, GA, Publication, AAV, Geographic atrophy, IVT, Protein, Patient, Complement factor I, Kaay Raav Tumhi, Annual general meeting, Cell, Retinal pigment epithelium, Sf9 (cells), Poster, RPE, Kinetics, Society, Disease, Vaccine, Chemistry, Adverum Biotechnologies

REDWOOD CITY, Calif., May 18, 2023 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today presented new nonclinical data on an IVT gene therapy for the treatment of GA secondary to dry age-related macular degeneration (dry AMD) via expression of CFI at the American Society of Gene & Cell Therapy (ASGCT) 2023 Annual Meeting, in Los Angeles, California. Adverum also presented new research pipeline data supporting the utility of its proprietary adeno-associated virus (AAV) vector platform in ocular gene therapy with presentations on optogenetics and on Chemistry, Manufacturing and Controls (CMC) strategies for AAV-mediated gene therapies in highly prevalent diseases.

Key Points: 
  • Adverum also presented new research pipeline data supporting the utility of its proprietary adeno-associated virus (AAV) vector platform in ocular gene therapy with presentations on optogenetics and on Chemistry, Manufacturing and Controls (CMC) strategies for AAV-mediated gene therapies in highly prevalent diseases.
  • “The ability to deliver both CFI and an engineered melanopsin leveraging our proprietary capsids via IVT administration demonstrates the strength of our platform at Adverum,” commented Brigit Riley, Ph.D., chief scientific officer of Adverum Biotechnologies.
  • “Similar to wet AMD, patients with GA are forced either to receive frequent, often monthly, injections or to experience faster lesion growth and vision deterioration.
  • The ASGCT poster and oral presentations will be made available on the Publications page of the Adverum website.