Stargardt

Ocugen to Present at BIO International Convention 2024

Retrieved on: 
onsdag, maj 29, 2024
3D, Patient, Armada, Exhibition, Booth, Clinical trial, Stargardt disease, Vaccine, Stargardt, BIO, Geographic atrophy, Retinitis pigmentosa, Cartilage, Pharmaceutical industry

MALVERN, Pa., May 29, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the Company will present at the 2024 BIO International Convention taking place in San Diego, CA from June 3 – 6, 2024.

Key Points: 
  • MALVERN, Pa., May 29, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the Company will present at the 2024 BIO International Convention taking place in San Diego, CA from June 3 – 6, 2024.
  • “We are excited to return to BIO this year and showcase the significant advancements we've achieved both clinically and strategically,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen.
  • “BIO is an important opportunity to pursue collaborations that will propel the development of our first-in-class therapies while also enhancing shareholder value.
  • Ocugen’s presentation details are as follows:
    Location: Presentation Theater 1 - Hall A - Exhibition Hall, San Diego Convention Center
    Please visit Ocugen at Booth #3945 to learn more about the Company’s clinical programs and the potential they may hold for patients.

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

Retrieved on: 
måndag, maj 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.

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

Retrieved on: 
onsdag, maj 8, 2024
UCLA, Stargardt disease, Stargardt, Research, Paratriathlon, Genetics, Retina, Patient, Human genetics, The central science, ABCA4, Clinical trial, Annual general meeting, ARVO, Vitamin A, Pharmaceutical industry

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.

Belite Bio Presents Additional Analysis from Phase 2 Study of Tinlarebant in Stargardt Disease at the ARVO Annual Meeting

Retrieved on: 
måndag, maj 6, 2024
Stargardt disease, University, Bias, Westmead, Stargardt, Research, Geographic atrophy, DRAGON, Adolescence, Growth, Observation, Disease, Therapy, Retina, Dry, Patient, Classification, CSO, Genotype, ABCA4, Annual general meeting, Belite, ARVO, SAN, Visual acuity, Pharmaceutical industry

Tinlarebant is Belite Bio’s orally administered tablet intended to slow disease progression in patients affected with STGD1 and Geographic Atrophy (GA) in advanced Dry Age-related Macular Degeneration (Dry AMD).

Key Points: 
  • Tinlarebant is Belite Bio’s orally administered tablet intended to slow disease progression in patients affected with STGD1 and Geographic Atrophy (GA) in advanced Dry Age-related Macular Degeneration (Dry AMD).
  • Genetic profiling was performed on the 13 adolescent STGD1 patients enrolled in a Phase 2 study of Tinlarebant.
  • “Data from the genotype and lesion growth analyses from our completed Phase 2 trial continues to demonstrate the efficacy of Tinlarebant,” said Dr. Nathan Mata, CSO of Belite Bio.
  • The presentation is now available in the “Presentations & Events” section of Belite Bio’s website: https://investors.belitebio.com/presentations-events/events.

AAVantgarde announces updated NHP data from its Stargardt disease program in a poster presentation at the ARVO 2024 annual meeting

Retrieved on: 
onsdag, april 24, 2024
Ophthalmology, Safety, Stargardt, Research, AAV, CSO, NHP, ABCA4, Stargardt disease, ARVO, Vaccine

MILAN, Italy, April 24, 2024 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage, Italian-based international biotechnology company with two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery, today announces the publication of an abstract submitted to The Association for Research in Vision and Ophthalmology Annual Meeting (ARVO), to be held May 5-9 in Seattle.

Key Points: 
  • MILAN, Italy, April 24, 2024 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage, Italian-based international biotechnology company with two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery, today announces the publication of an abstract submitted to The Association for Research in Vision and Ophthalmology Annual Meeting (ARVO), to be held May 5-9 in Seattle.
  • “We are delighted to be presenting positive NHP safety and efficacy data from our Stargardt program demonstrating that AAV.ABCA4.intein vectors can be administered safely to NHPs under an immunosuppressive protocol designed for use in man, and at doses showing robust expression matching the pattern of endogenous ABCA4.
  • It is encouraging to see that our AAV.ABCA4.intein product shows potential to treat retinal dystrophies, such as Stargardt Disease,” said Prof. Alberto Auricchio, CSO of AAVantgarde.
  • Dr. Natalia Misciattelli, CEO of AAVantgarde explained that “This positive data in NHP is very encouraging as it shows the potential of our Stargardt program, and it provides hope to the underserved Stargardt patients that currently don’t have many therapeutic options available.”
    Presentation Number / Posterboard Number: 6097 - B0943

AAVantgarde presents positive preclinical data in large animal models from its Stargardt disease program in an oral presentation at the ASGCT 2024 annual meeting

Retrieved on: 
tisdag, april 23, 2024
Stargardt disease, Safety, Stargardt, Cell, AAV, Messenger, Lipofuscin, Gene, Society, CSO, NHP, Vaccine

The data to be presented confirms that AAV intein-mediated retinal gene therapy for Stargardt disease is effective and safe in large animal models (pig and NHP).

Key Points: 
  • The data to be presented confirms that AAV intein-mediated retinal gene therapy for Stargardt disease is effective and safe in large animal models (pig and NHP).
  • In pigs, AAVantgarde demonstrate that lipofuscin accumulation in the retinal pigmented epithelium was reduced upon subretinal delivery of AAV-ABCA4 intein vectors.
  • Similarly, in NHP, BaseScope analysis showed nearly total photoreceptor co-expression of mRNAs encoding both ABCA4-intein halves across an extended NHP retinal region.
  • Prof. Alberto Auricchio, CSO of AAVantgarde stated “We are very excited to be presenting positive Pig and NHP safety and efficacy data from our Stargardt program as an oral presentation, supporting both the efficacy and safety of AAV-ABCA4-intein vectors in relevant large animal models, and providing important insights towards the clinical translation of this platform for gene therapy of STGD1.”

Nanoscope Therapeutics Enhances Mutation-Independent Retinal Gene Therapy Programs with Appointment of Allen C. Ho, MD, as Chief Medical Advisor

Retrieved on: 
torsdag, april 25, 2024
Stargardt disease, RP, State, Stargardt, Research, Diabetic retinopathy, Communication, FACS, Thomas Jefferson University, Patient, Geographic atrophy, American Academy of Ophthalmology, Clinical trial, Therapy, AAO, Medical device

DALLAS, April 25, 2024 /PRNewswire/ -- Nanoscope Therapeutics, Inc. , a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, announced today the appointment of Allen C. Ho, MD, FACS, FASRS, as Chief Medical Advisor, where he will help define the strategy of developing the gene mutation-agnostic therapies.

Key Points: 
  • DALLAS, April 25, 2024 /PRNewswire/ -- Nanoscope Therapeutics, Inc. , a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, announced today the appointment of Allen C. Ho, MD, FACS, FASRS, as Chief Medical Advisor, where he will help define the strategy of developing the gene mutation-agnostic therapies.
  • Dr. Ho is Attending Surgeon and Director of Retina Research at Wills Eye Hospital and Co-Director of the Wills Eye Hospital Retina Service.
  • "We are excited to have Dr. Allen C. Ho join Nanoscope as Chief Medical Advisor," said Sulagna Bhattacharya, Nanoscope co-founder and CEO.
  • "As a world-renowned retina specialist and leader in innovative treatments for retinal diseases, his insight will be invaluable to Nanoscope.

Prevent Blindness Declares May as First-ever Inherited Retinal Disease (IRD) Genetic Testing Awareness Month

Retrieved on: 
onsdag, april 24, 2024
Education, Paratriathlon, University, Blood, Birth defect, Retina, RP, Social media, Diagnosis, Stargardt disease, Stargardt, Marketing, Strabismus, Genetic, Johnson & Johnson, Gene, Spark Therapeutics, Choroid, Research, Visual impairment, Genetic testing, Pediatric ophthalmology, Disease, Visual Science, Patient, Eye, Cone dystrophy, MBA, Vision, Student, Therapy, Organization, IRD, Pharmaceutical industry

CHICAGO, April 24, 2024 /PRNewswire-PRWeb/ -- Prevent Blindness, the nation's leading nonprofit eye health and safety organization, has designated May as "Inherited Retinal Disease (IRD) Genetic Testing Awareness Month," expanding its previous IRD and Genetic Testing Awareness Week initiative. The group is providing a variety of tools to promote awareness and education for IRDs and the importance of genetic testing, including a free webinar, expert and patient videos, shareable social media graphics, and fact sheets in English and Spanish. IRD Genetic Testing Awareness Month is supported by funding from Johnson & Johnson, and Spark® Therapeutics.

Key Points: 
  • Prevent Blindness expands IRD and genetic testing awareness initiative to educate patients on various forms of IRDs, and the importance of genetic testing to confirm diagnosis and possible treatments.
  • CHICAGO, April 24, 2024 /PRNewswire-PRWeb/ -- Prevent Blindness , the nation's leading nonprofit eye health and safety organization, has designated May as "Inherited Retinal Disease (IRD) Genetic Testing Awareness Month," expanding its previous IRD and Genetic Testing Awareness Week initiative.
  • ET, the National Center for Children's Vision and Eye Health at Prevent Blindness will be hosting the free webinar, " Pathway to Diagnosis: Genetic Testing for Inherited Retinal Diseases ."
  • To register for the "Pathway to Diagnosis: Genetic Testing for Inherited Retinal Diseases" webinar, visit PreventBlindness.org/IRD-webinar-2024.

US Retinal Specialists Highlight the Greatest Opportunity for Gene Therapies

Retrieved on: 
tisdag, mars 19, 2024
Retina, Stargardt disease, Macular edema, Retinitis pigmentosa, Eye, Intelligence, Paratriathlon, Hope, Stargardt, Adverum Biotechnologies, Safety, NAMD, Geographic atrophy, Ophthalmology, Diabetic retinopathy, Medical device

Unlike other organs, the eye's compact size and accessibility have made it an ideal application for gene therapies, offering targeted treatment with minimal systemic impact.

Key Points: 
  • Unlike other organs, the eye's compact size and accessibility have made it an ideal application for gene therapies, offering targeted treatment with minimal systemic impact.
  • As advancements continue, a plethora of gene therapies for retinal disorders are on the horizon.
  • Spherix Global Insights collaborated with 77 retinal specialists to understand their perspectives on adopting gene therapies, specifically where specialists were and were not willing to use gene therapy across diseases and patient types.
  • While retinal specialists express eagerness for gene therapies, dissatisfaction towards current treatments like Syfovre and Izervay underscores the need for innovation.

Nanoscope Therapeutics Announces Participation in Upcoming Conferences

Retrieved on: 
onsdag, februari 21, 2024
Stargardt, Retina, Stargardt disease, BIO, Therapy, AMD, Patient, Pharmaceutical industry

DALLAS, Feb. 21, 2024 /PRNewswire/ -- Nanoscope Therapeutics Inc. , a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced that Sulagna Bhattacharya, Chief Executive Officer of Nanoscope Therapeutics, and other members of its senior management team will participate in two upcoming conferences.

Key Points: 
  • DALLAS, Feb. 21, 2024 /PRNewswire/ -- Nanoscope Therapeutics Inc. , a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced that Sulagna Bhattacharya, Chief Executive Officer of Nanoscope Therapeutics, and other members of its senior management team will participate in two upcoming conferences.
  • Details are as follows:
    Ms. Bhattacharya presently serves on the board of directors of BIO.
  • In her presentation, she will give an overview of Nanoscope's Multi-Characteristic Opsin platform and highlight the Company's gene-agnostic approach.
  • Nanoscope leadership will also be available for 1x1 meetings for the duration of the conference.