Natural history study

National Organization for Rare Disorders Launches Education Series to Advance Patient Involvement in Rare Disease Drug Development

Retrieved on: 
Monday, October 16, 2023

WASHINGTON, Oct. 16, 2023 /PRNewswire/ -- Today, the National Organization for Rare Disorders (NORD®) launched a new education series in English and Spanish titled, "Rare Disease Drug Development: What Patients and Advocates Need to Know," designed to help patients and patient advocacy groups understand the drug development process.

Key Points: 
  • WASHINGTON, Oct. 16, 2023 /PRNewswire/ -- Today, the National Organization for Rare Disorders (NORD®) launched a new education series in English and Spanish titled, "Rare Disease Drug Development: What Patients and Advocates Need to Know," designed to help patients and patient advocacy groups understand the drug development process.
  • New education series in English and Spanish: "Rare Disease Drug Development: What Patients and Advocates Need to Know.
  • "The goal of this educational series is to engage the rare disease community on their essential role in the drug development process and ultimately help produce more and better treatments for rare disease," said Rebecca Aune, Director of Education Programs at the National Organization for Rare Disorders.
  • The first modules, on "Drug Development Process", "Stakeholder Roles in Drug Development" and "Natural History Studies," are now available for free at learn.rarediseases.org .

 Rallybio Announces Proof-of-Concept Results and Development Updates for RLYB212, a Novel Monoclonal anti-HPA-1a Antibody to Prevent Fetal and Neonatal Alloimmune Thrombocytopenia

Retrieved on: 
Saturday, June 24, 2023

Mean platelet elimination half-life was 5.8 hours (0.09mg) and 1.5 hours (0.29mg) for RLYB212 compared to 71.7 hours for placebo.

Key Points: 
  • Mean platelet elimination half-life was 5.8 hours (0.09mg) and 1.5 hours (0.29mg) for RLYB212 compared to 71.7 hours for placebo.
  • The study included 11 males aged 18 to 65 years, randomized to RLYB212 0.09mg (n=4), RLYB212 0.29mg (n=5), or placebo (n=2).
  • Rallybio will host an investor and analyst meeting on Saturday, June 24, 2023 from 4:00 to 6:00 p.m. Eastern Time.
  • The webcast and corresponding slides can be accessed through the Events and Presentations section of Rallybio’s website at http://www.rallybio.com .

Benitec Biopharma to Present at the OPMD International Conference

Retrieved on: 
Monday, May 8, 2023

HAYWARD, Calif., May 08, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced that Jerel A.

Key Points: 
  • HAYWARD, Calif., May 08, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced that Jerel A.
  • Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec will present virtually on the BB-301 Clinical Development Program at the Oculopharyngeal Muscular Dystrophy (OPMD) International Conference in Tel Aviv, Israel, on Tuesday, May 16th, 2023 at 12:50 pm Israel Daylight Time.
  • Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec.
  • “Enrollment of OPMD subjects into the Natural History Study at the U.S. clinical trial site is proceeding at a rapid pace, with 9 subjects enrolled to date.

Sensorion Announces Candidate Selection for GJB2 Gene Therapy Program

Retrieved on: 
Thursday, April 6, 2023

Géraldine Honnet, M.D., Chief Medical Officer of Sensorion, said: “We are very proud to have selected a candidate for our GJB2-GT program.

Key Points: 
  • Géraldine Honnet, M.D., Chief Medical Officer of Sensorion, said: “We are very proud to have selected a candidate for our GJB2-GT program.
  • “Sensorion believes gene therapy has the potential to transform lives and we are delighted to be moving this promising candidate forward towards clinical development.
  • Sensorion also plans to file a Clinical Trial Application for its OTOF-GT program, a dual-vector AAV gene therapy program for the treatment of children born with hearing loss caused by otoferlin deficiency this quarter.
  • Christine Petit
    GJB2-GT Program: Data to Drive Next Steps, Dr. Laurent Désiré
    GJB2-GT Program: Natural History Studies to Prepare Execution of Clinical Trials, Dr. Géraldine Honnet
    OTOF-GT Program: SENS-501, Sensorion’s Lead Gene Therapy Program, Dr. Laurent Désiré
    Enabling Reliable Gene Therapy Manufacturing and Analytical Control, Dr. Christine Le Bec

Inozyme Pharma Announces Investor and Analyst Event and Highlights 2022 Progress

Retrieved on: 
Monday, January 9, 2023

BOSTON, Jan. 09, 2023 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced that it will share topline pharmacokinetic, pharmacodynamic (PK/PD) and safety data from the ongoing Phase 1/2 clinical trials of INZ-701 in ENPP1 Deficiency and ABCC6 Deficiency at a virtual Investor and Analyst Event on Thursday, Feb. 16, 2023.

Key Points: 
  • We also saw promising PPi elevation in our ABCC6 Deficiency trial, with a rapid initial increase at the lowest dose of INZ-701.
  • The Company will share topline pharmacokinetic, pharmacodynamic (PK/PD) and safety data from ongoing Phase 1/2 trials of INZ-701 in ENPP1 Deficiency and ABCC6 Deficiency.
  • The event will also feature presentations from members of the Inozyme management team, as well as from key opinion leaders in ENPP1 Deficiency and ABCC6 Deficiency.
  • The webcast will be accessible through the Investor Relations section of Inozyme’s website under events and will be available for a limited time following the event.

Khondrion announces sonlicromanol Phase IIb progress supporting Phase III development in MELAS spectrum disorders

Retrieved on: 
Tuesday, November 22, 2022

Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.

Key Points: 
  • Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.
  • In line with previous studies, sonlicromanol was found to be safe and well tolerated in the Phase IIb programme, with no serious adverse effects.
  • These longer-term patient data are, therefore, instrumental in providing valuable insights for the design of the upcoming Phase III trial.
  • One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has recently completed a Phase IIb study in adult patients with m.3243A>G MELAS spectrum disorders.

Khondrion announces sonlicromanol Phase IIb progress supporting Phase III development in MELAS spectrum disorders

Retrieved on: 
Tuesday, November 22, 2022

Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.

Key Points: 
  • Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.
  • In line with previous studies, sonlicromanol was found to be safe and well tolerated in the Phase IIb programme, with no serious adverse effects.
  • These longer-term patient data are, therefore, instrumental in providing valuable insights for the design of the upcoming Phase III trial.
  • One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has recently completed a Phase IIb study in adult patients with m.3243A>G MELAS spectrum disorders.

eClinical Solutions Thought Leaders to Discuss Managing Evolution of Clinical Data, Standards, and Technology at SCDM, DPHARM, CDISC, Among Other Upcoming Industry Conferences

Retrieved on: 
Thursday, September 8, 2022

eClinical Solutions will also be presenting and exhibiting at DPHARM 2022 (September 13-14) in Boston, MA.

Key Points: 
  • eClinical Solutions will also be presenting and exhibiting at DPHARM 2022 (September 13-14) in Boston, MA.
  • The eClinical Solutions team will also be available in booth #48a of the exhibit hall to demonstrate elluminate.
  • eClinical Solutions will close out the month of October at the 2022 Clinical Data Interchange Standards Consortium (CDISC) US Interchange (October 26-27) in Austin, TX.
  • eClinical Solutions helps life sciences organizations around the world accelerate their digital clinical initiatives with modern data infrastructure and analytics.

Vigil Neuroscience Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Business Highlights

Retrieved on: 
Friday, March 25, 2022

CAMBRIDGE, Mass., March 25, 2022 (GLOBE NEWSWIRE) -- Vigil Neuroscience, Inc. (Nasdaq: VIGL), a clinical-stage biotechnology company committed to harnessing the power of microglia for the treatment of neurodegenerative diseases, today announced financial results for the fourth quarter and full year ended December 31, 2021 and provided an update on recent business highlights.

Key Points: 
  • The gross proceeds of the offering were $98 million, before deducting underwriting discounts and commissions and other offering expenses.
  • 11,186,636, which describes the VGL101 antibody and claims compositions of matter, pharmaceutical compositions and methods of production, each directed to the VGL101 antibody.
  • This underpins the Companys anti-TREM2 antibody technology and provides Vigil with intellectual property protection.
  • As a result, 2020 financial reporting below represents only partial year results vs 2021 reporting which reflects 12 full months of operations.

Sensorion Reports 2021 First Half Results

Retrieved on: 
Monday, September 27, 2021

Many elements of Sensorions strategic planning came together in a successful first half of 2021.

Key Points: 
  • Many elements of Sensorions strategic planning came together in a successful first half of 2021.
  • Since not all of the patients have yet completed their 28-day end of treatment visit, Sensorion will continue the recruitment until the end of October 2021.
  • Following this initial success, Sensorion and Cochlear announced the initiation of a Phase 1 clinical trial of SENS-401 (Arazasetron) in patients scheduled for cochlear implantation on September 8, 2021.
  • The study will be sponsored by Sensorion and the proposed design is expected to be submitted to regulatory authorities during H2 2021 (see post-closing events).