GM2

Azafaros announces completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients

Retrieved on: 
Tuesday, March 12, 2024
Pharmacokinetics, Niemann–Pick disease, Hospital, NPC, GM2, Patient, RAINBOW, Lead, Safety, Pharmaceutical industry

Leiden, The Netherlands, March 12, 2024 – Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW ( Phase 2 RAINBOW study NCT05758922 ).

Key Points: 
  • Leiden, The Netherlands, March 12, 2024 – Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW ( Phase 2 RAINBOW study NCT05758922 ).
  • The randomized, double-blind, placebo-controlled study, conducted in Brazil, involved 13 patients from the age of 12 years who are affected by GM2 gangliosidosis or Niemann-Pick disease type C (NPC).
  • The aim of the RAINBOW study is to determine the safety, pharmacodynamics, and pharmacokinetics of two different doses of nizubaglustat in patients, in order to identify the target dosage for Azafaros’ planned Phase 3 pivotal studies.
  • Azafaros is grateful to the patients and their families who made the decision to participate in this study.”

Taysha Gene Therapies Provides Update on Deprioritized Pipeline Programs

Retrieved on: 
Thursday, February 15, 2024
NINDS, Central nervous system, Rush University Medical Center, Giant axonal neuropathy, GM2, Rett syndrome, CNS, IP, GAN, IND, AAV, DSM-IV codes, Pharmaceutical industry

DALLAS, Feb. 15, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha” or “the Company”), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today provided an update on its deprioritized pipeline programs as part of an ongoing effort to help support their further potential development.

Key Points: 
  • DALLAS, Feb. 15, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha” or “the Company”), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today provided an update on its deprioritized pipeline programs as part of an ongoing effort to help support their further potential development.
  • Taysha has been working to find ways to advance its deprioritized programs.
  • On November 13, 2023, Taysha terminated its existing loan and security agreement and entered into a new loan and security agreement that provides consent to allow the Company to transfer intellectual property (IP) for several deprioritized programs to third parties in a more efficient manner.
  • “Today’s announcement demonstrates meaningful progress to advance important development work for several deprioritized programs.

National Tay-Sachs & Allied Diseases Association Hosts First of Its Kind Drug Development Meeting for GM2

Retrieved on: 
Thursday, February 1, 2024
GM2, Parent, DSM-IV codes, GM1, Tay–Sachs disease, Caregiver, Drug development, Patient, FDA, Marketing, National Institutes of Health, Food, Disease, Bangladesh Technical Education Board, Doctor of Philosophy, Infant, Pharmaceutical industry

BOSTON, Feb. 1, 2024 /PRNewswire/ -- National Tay-Sachs & Allied Diseases Association (NTSAD), leader in the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases, is hosting the first-ever, Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) on February 15, 2024, 9:30am-3pm Eastern.

Key Points: 
  • BOSTON, Feb. 1, 2024 /PRNewswire/ -- National Tay-Sachs & Allied Diseases Association (NTSAD), leader in the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases, is hosting the first-ever, Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) on February 15, 2024, 9:30am-3pm Eastern.
  • This input can inform FDA's decisions and oversight during drug development and their review of marketing applications.
  • Nearly 30 people will share their stories and experiences with infantile, juvenile, or late onset GM2, including adult patients, parents, caregivers, and bereaved family members.
  • This virtual meeting will be livestreamed via YouTube, open to the public, free to attend, but advance registration is required.

Azafaros to present data from PRONTO study in patients with GM1 and GM2 gangliosidoses at the 20th annual WORLDSymposium™

Retrieved on: 
Tuesday, January 23, 2024
RAINBOW, NPC, Glycogen storage disease, GM2, Patient, Research, GM1, Niemann–Pick disease, Gait, Science, Sleep disorder, Molecular genetics, Gangliosidosis, Safety, Central nervous system, Congress, Biomarker, Pharmacokinetics, Metabolism, Pharmaceutical industry

As well as data from PRONTO, Azafaros will present details of its Phase 2 RAINBOW study, aimed at investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat in patients with GM2 gangliosidosis or NPC.

Key Points: 
  • As well as data from PRONTO, Azafaros will present details of its Phase 2 RAINBOW study, aimed at investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of nizubaglustat in patients with GM2 gangliosidosis or NPC.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO) - patients’ and caregivers’ assessments.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO)- baseline clinical data.
  • Title: A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO) - evaluation of different assessments.

Orphan designation: (2S,3R,4R,5S)-1-[5-(2-Fluoro-biphenyl-4-ylmethoxy)-pentyl]-2-hydroxymethyl-piperidine-3,4,5-triol Treatment of GM2 gangliosidosis, 15/02/2023 Positive

Retrieved on: 
Saturday, January 20, 2024
COMP, Committee, GM2, European Commission, IRIS, EMA, Pharmaceutical industry

EU/3/23/2762 - orphan designation for treatment of GM2 gangliosidosis

Key Points: 
  • EU/3/23/2762 - orphan designation for treatment of GM2 gangliosidosis
    (2S,3R,4R,5S)-1-[5-(2-Fluoro-biphenyl-4-ylmethoxy)-pentyl]-2-hydroxymethyl-piperidine-3,4,5-triol
    OrphanHuman
    Azafaros B.V.
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled

Retrieved on: 
Monday, December 11, 2023
Patient, NPC, GM1, GM2, Lead, MD, Glycogen storage disease, Clinical trial, Pharmacokinetics, DSM-IV codes, GM2 gangliosidoses, Niemann–Pick disease, Safety, RAINBOW, Pharmaceutical industry

The success in enrollment is a major milestone for Azafaros’ lead asset, nizubaglustat, which is being developed to treat patients with GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC).

Key Points: 
  • The success in enrollment is a major milestone for Azafaros’ lead asset, nizubaglustat, which is being developed to treat patients with GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC).
  • The 12-weeks study is a randomised, double-blind, placebo-controlled, multi-center trial which evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics across two doses of nizubaglustat in patients with GM2 gangliosidosis and NPC.
  • The trial is being conducted at sites across Brazil, with safety, pharmacokinetic and pharmacodynamic data from ongoing patient follow-ups expected in mid-2024.
  • We would like to thank all patients, their families, patient organisations and clinicians for their participation and support in this important research.”

Bild Expo Is Live: Join Us at Gear Expo for New Product Announcements and Educational Presentation

Retrieved on: 
Wednesday, September 6, 2023
Hardware, Photography, Audio, Video, Consumer Electronics, Technology, Maker, Eclipse, Carbon, Well, MT, Javits Center, Life, APS, ENG, GoPro, Summicron, B&H, Special edition, Temperature, Canon RF lens mount, ARRI, Titanium, II, Projection, MacOS, Sonos, SanDisk, Collection, Bluetooth, Sport, Data-rate units, Transport, Leica, FIZ, L3, COB, CFexpress, Lowepro, TB, EF, Element, Nanook, LED, RAID 1, Barndoor skate, Specific, Iris, Canon EF lens mount, Carbon-fiber-reinforced polymers, GM2, EA, Shape, CCT, Multimedia, AC, ANSI, Tablet, Gold, Sigma, Hertz, CompactFlash, Traveller Alien Module 8: Darrians, Lensbaby, L1, Light-emitting diode, Sony, Lens, Super 35, Traveler, SKB, Corrosion, Ecosystem, RGB, BIONZ, Television, Computer data storage, Camera, Video game, Telescopic sight, Cassette tape, Daystar, L2, T16

Angelbird’s other offering for B&H’s 50th-anniversary celebration is the 512GB AV PRO CFexpress 2.0 Type B SE Memory Card .

Key Points: 
  • Angelbird’s other offering for B&H’s 50th-anniversary celebration is the 512GB AV PRO CFexpress 2.0 Type B SE Memory Card .
  • Designer of high-end cine lenses from France, Angenieux has announced the EZ-3 Series Convertible Super 35 and Full-Frame/VistaVision zoom lens.
  • Maker of ergonomic carrying solutions for long shoots, BlackRapid is releasing the special 50th-anniversary-edition Curve Breathe Camera Strap Bundle .
  • The bundle includes a curve breathe camera strap, an underarm stabilizer, a safety tether II, a Lockstar II, and two FR-5 FastenRs.

New Hope Research Foundation and Forge Biologics Announce cGMP Manufacturing Partnership to Accelerate Gene Therapy for Patients with Tay-Sachs Disease

Retrieved on: 
Wednesday, June 28, 2023
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Ignition, Partnership, GM2, Glycogen storage disease, Hope, Multimedia, AAV, Forge, Tay–Sachs disease, CDMO, CGMP, Patient, Entrepreneurship, HEK293, Pharmaceutical industry, Hearth

View the full release here: https://www.businesswire.com/news/home/20230628556153/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20230628556153/en/
    “We are proud to be the CDMO partner for New Hope Research Foundation, an organization that is significantly advancing a gene therapy approach to provide hope for patients suffering from Tay-Sachs disease,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge Biologics.
  • “At Forge, we are ready to partner with companies, nonprofit organizations, and gene therapy developers around the world, working towards the mission we share to bring potentially life-saving gene therapies to patients.”
    Through this partnership, Forge will provide adeno-associated virus (AAV) process development, analytical services, and cGMP manufacturing to New Hope Research Foundation.
  • The Foundation will leverage Forge’s platform processes, including its proprietary HEK293 suspension Ignition Cells™, to accelerate the initial production.
  • All development and AAV manufacturing activities will occur at the Hearth, Forge’s 200,000 square foot gene therapy facility in Columbus, Ohio.

Azafaros Announces Enrollment of First Patient in Phase 2 RAINBOW Study Evaluating AZ-3102 in GM2 and NP-C Patients

Retrieved on: 
Thursday, June 8, 2023
Pharmaceutical, Health, Clinical Trials, RAINBOW, Lead, NP-C, GM2, Clinical trial, Enzyme, Pharmacokinetics, GM1, Gangliosidosis, DSM-IV codes, Metabolism, Patient, Niemann–Pick disease, NP, Diagnosis, Hospital, Glycosphingolipid, Safety, Pharmaceutical industry

Azafaros B.V. today announced that the first patient has been enrolled into its Phase 2 RAINBOW study ( NCT05758922 ).

Key Points: 
  • Azafaros B.V. today announced that the first patient has been enrolled into its Phase 2 RAINBOW study ( NCT05758922 ).
  • Azafaros is developing the compound as a potentially disease-modifying treatment in severe metabolic disorders including GM1 and GM2 gangliosidoses and NP-C. Enrollment of the first patient in the study is an important milestone for Azafaros in its mission to bring new treatment options to these patients and their families.
  • The Phase 2 RAINBOW study is a randomized, double-blind, placebo-controlled, multicenter, 12-week trial assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of AZ-3102 in patients with GM2 gangliosidosis and NP-C.
  • Patients who complete the 12-week study period will be offered a double-blind extension if approved by the country’s health authorities.

Azafaros Appoints Chief Operating Officer and Head of Finance

Retrieved on: 
Monday, March 20, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, RAINBOW, Drug development, GM2, Head, Nordic, Strategic planning, Doctor of Philosophy, Novo Holdings A/S, Novartis, Patient, Niemann–Pick disease, Series, Commercial, Rare, Pharmaceutical industry, Oncology, Stargazer

Silvia Ragno, PhD, joins the company as Chief Operating Officer and Henrik Torp Nielsen as Head of Finance.

Key Points: 
  • Silvia Ragno, PhD, joins the company as Chief Operating Officer and Henrik Torp Nielsen as Head of Finance.
  • Her primary responsibility as COO will be strategic planning and execution of our investor relations and business development efforts,” said Stefano Portolano, Chief Executive Officer of Azafaros.
  • “Henrik has previously occupied diverse finance roles in numerous countries and is a formidable addition to our team as Head of Finance.
  • He previously held several senior roles at Novartis, notably European Head of Business Development Finance, Oncology, as well as Nordic Chief Financial Officer, Oncology and Head of Commercial and Medical Finance, successfully supporting and closing several major business development deals.