Paratriathlon

World's Most Influential People in Ophthalmology

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четверг, мая 9, 2024

MIAMI, May 9, 2024 /PRNewswire/ -- Fifteen Bascom Palmer Eye Institute physicians were selected for the 2024 Power List, a prestigious selection of the world's 100 most influential people shaping the future of eye care, as published by The Ophthalmologist magazine. Drs. Eduardo C. Alfonso, Kendall Donaldson, Hilda Capó, Janet Davis, Harry Flynn, Anat Galor, Steven Gedde, Ranya Habash, Carol Karp, Jaclyn Kovach, Byron Lam, Felipe Medeiros, Richard Parrish, Philip Rosenfeld, and Sonia Yoo were named to the 2024 Power List. Bascom Palmer Eye Institute is the Department of Ophthalmology for the University of Miami Miller School of Medicine and UHealth – University of Miami Health System.

Key Points: 
  • Bascom Palmer Eye Institute is the Department of Ophthalmology for the University of Miami Miller School of Medicine and UHealth – University of Miami Health System.
  • From cutting-edge treatments to groundbreaking research, these doctors are pushing boundaries and setting new standards in ophthalmology."
  • Alfonso, the holder of the Kathleen and Stanley J. Glaser Chair in Ophthalmology, was also named to the Ophthalmologist Power Lists 2014, 2016, 2018, 2022, and 2023.
  • Holder of the John T. Flynn Chair in Ophthalmology, Hilda Capó, M.D., specializes in pediatric ophthalmology and adult strabismus.

Surrozen Presents Data Demonstrating the Promise of Antibody-Based Wnt Mimetics in Treating Cornea Endothelial Dystrophies and Dry Eye Disease at the Associations for Research in Vision and Ophthalmology (ARVO) Annual Meeting

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четверг, мая 9, 2024

“These early results are important as Fuchs’ Endothelial Cell Dystrophy and Dry Eye Disease are severe diseases with few effective treatments available.

Key Points: 
  • “These early results are important as Fuchs’ Endothelial Cell Dystrophy and Dry Eye Disease are severe diseases with few effective treatments available.
  • The company evaluated a therapeutic approach to stimulate endothelial cell proliferation as human corneal endothelial cells have a limited capacity to regenerate.
  • The lead molecule, a Surrozen Fzd 1/2/7 SWAP antibody, increased proliferation of endothelial cells in vitro in human cells.
  • Currently, there is no epithelial regeneration strategy available as treatment usually consists of anti-inflammatory topical eye drops and tear replacements.

Alkeus Pharmaceuticals Announces Positive Interim Results Demonstrating No Signs of Disease Progression in Early-Stage Stargardt Disease Patients Treated with Gildeuretinol

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среда, мая 8, 2024

CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc. today announced the presentation of positive interim data from its TEASE-3 study demonstrating that early-stage Stargardt disease patients treated with gildeuretinol acetate showed no disease progression and remained asymptomatic for the duration of therapy ranging between two and six years.

Key Points: 
  • CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc. today announced the presentation of positive interim data from its TEASE-3 study demonstrating that early-stage Stargardt disease patients treated with gildeuretinol acetate showed no disease progression and remained asymptomatic for the duration of therapy ranging between two and six years.
  • Results were presented during the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting being held May 5-9 in Seattle.
  • “These exciting results demonstrate the potential of gildeuretinol acetate to prevent vision loss in early-stage Stargardt patients,” said Leonide Saad, Ph.D., President and CEO of Alkeus Pharmaceuticals.
  • All of us working in our growing Alkeus team are committed to advancing research and having an impact on patients living with vision loss.

CRISPR Therapeutics Highlights ASGCT Oral Presentation and Announces New Programs Utilizing In Vivo Gene Editing Approach

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среда, мая 8, 2024

In addition, CRISPR Therapeutics announced the expansion of its in vivo pipeline with two new programs.

Key Points: 
  • In addition, CRISPR Therapeutics announced the expansion of its in vivo pipeline with two new programs.
  • “The expansion of our in vivo pipeline speaks to the scalability of the platform and the exceptional translation capabilities of our team.
  • The addition of two more programs, CTX340 and CTX450, utilizing this LNP delivery technology demonstrates the modularity and scalability of the platform.
  • In today’s presentation, the Company presented data demonstrating efficient and specific delivery to TM cells in mouse, non-human primate, and ex vivo human eyes.

Editas Medicine Announces First Quarter 2024 Results and Business Updates

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среда, мая 8, 2024

CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, today reported financial results for the first quarter 2024 and provided business updates.

Key Points: 
  • CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, today reported financial results for the first quarter 2024 and provided business updates.
  • “We made significant progress in all three pillars of our strategy this quarter, including reni-cel, in vivo, and business development, including intellectual property,” commented Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine.
  • ET to provide and discuss a corporate update and financial results for the first quarter of 2024.
  • To access the call, please dial 1-877-407-0989 (domestic) or 1-201-389-0921 (international) and ask for the Editas Medicine earnings call.

Oculis Announces Completion of Enrollment in Phase 2 Randomized Controlled ACUITY Trial with OCS-05 for Acute Optic Neuritis

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среда, мая 8, 2024

The Phase 2 ACUITY study is evaluating once-daily OCS-05 intravenous infusion in patients with AON.

Key Points: 
  • The Phase 2 ACUITY study is evaluating once-daily OCS-05 intravenous infusion in patients with AON.
  • OCS-05 has been granted orphan drug designation in both the United States and Europe, highlighting the importance of this unmet medical need.
  • In the meantime, we keep on working towards an IND submission for OCS-05 in the U.S. in 2024.
  • While corticosteroids are used to shorten the attack, there is no approved therapy for AON and unmet needs remain for therapies that can prevent vision loss after an acute episode of optic neuritis.

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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вторник, мая 7, 2024

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.

EyeDNA Therapeutics Announces Positive 24-month Data Presented at ARVO from Ongoing Phase I/II Trial of HORA-PDE6b Gene Therapy in Patients with Retinitis Pigmentosa Caused by Bi-allelic Mutations in PDE6b

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вторник, мая 7, 2024

These data were reported during an oral presentation* on May 6, at the Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting in Seattle, WA, US.

Key Points: 
  • These data were reported during an oral presentation* on May 6, at the Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting in Seattle, WA, US.
  • The positive 24-month follow-up data presented confirm results from the previous interim analysis of the trial conducted at the 12-month follow-up point and support preparation for a registrational trial for HORA-PDE6b in PDE6b RP patients.
  • Further discussions with health authorities in the US and Europe are planned to define the optimal path to making HORA-PDE6b available to PDE6b RP patients.
  • The treatment was administered in the more affected eye while the other eye served as an untreated control.

BioCryst Reports First Quarter 2024 Financial Results and Provides Business Update

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понедельник, мая 6, 2024

RESEARCH TRIANGLE PARK, N.C., May 06, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today reported financial results for the first quarter ended March 31, 2024, and provided a corporate update.

Key Points: 
  • These two successes helped us exceed our revenue projections for the quarter and, as a result, we are increasing our revenue guidance for 2024,” said Charlie Gayer, chief commercial officer of BioCryst.
  • Interest expense was $24.5 million in the first quarter of 2024, compared to $27.4 million in the first quarter of 2023 (-10.6 percent y-o-y).
  • Our references to our first quarter 2024 “non-GAAP pro forma” financial measures of adjusted net loss and adjusted earnings per share constitute non-GAAP financial measures.
  • ET today to discuss the financial results and provide a corporate update.

Boehringer Ingelheim shares positive results from the first study worldwide in diabetic macular ischemia

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понедельник, мая 6, 2024

Boehringer Ingelheim today presented positive data from the HORNBILL Phase I/IIa study of BI 764524, the first ever study exploring a potential treatment for people living with diabetic macular ischemia (DMI).

Key Points: 
  • Boehringer Ingelheim today presented positive data from the HORNBILL Phase I/IIa study of BI 764524, the first ever study exploring a potential treatment for people living with diabetic macular ischemia (DMI).
  • The current standard of care for advanced DR includes intravitreal anti-VEGF treatment or invasive laser treatment.
  • “Retinal non-perfusion is a key driver of vision loss in people living with diabetic retinopathy.
  • However, until the HORNBILL study, retinal non-perfusion has not been explored as a potential treatment target.”
    “Vision loss associated with retinal conditions such as diabetic retinopathy and DMI has a devastating impact on quality of life.