Ion channels

Ambry Genetics Leads in Clinical Contributions to GenCC, a Public Database to Inform the Relationship Between Various Genes and Diseases

Retrieved on: 
Tuesday, July 13, 2021

"We know our contribution to this data is vital in providing the right diagnosis to healthcare providers and their patients," said Tom Schoenherr, Chief Executive Officer at Ambry Genetics.

Key Points: 
  • "We know our contribution to this data is vital in providing the right diagnosis to healthcare providers and their patients," said Tom Schoenherr, Chief Executive Officer at Ambry Genetics.
  • "In addition, these advancements will assist in shaping our technological platforms CARE and LATTICE by providing relevant data to make informed healthcare and therapy decisions.
  • Ambry has pioneered the discovery of several gene-disease relationships, including the KCNN2 gene.
  • These gene mutations are now known to cause a KCNN2-related neurodevelopmental disorder which commonly presents with tremor and extrapyramidal movements.

Regulus Therapeutics Presents Additional Data from its Autosomal Dominant Polycystic Kidney Disease (ADPKD) Program at PKD Connect 2021

Retrieved on: 
Friday, June 25, 2021

Levels of PC1 and PC2 have previously been shown to be inversely correlated with disease severity.

Key Points: 
  • Levels of PC1 and PC2 have previously been shown to be inversely correlated with disease severity.
  • The poster also describes new data from relevant preclinical models showing treatment with RGLS4326 results in increased gene and polycystin levels in vitro.
  • In addition,improvements in key disease markers including serum creatinine and BUN were demonstrated in the Pkd1(F/RC) mouse model that harbors Pkd1 mutation equivalent to human ADPKD.
  • These and other risks are described in additional detail in Regulus' filings with the Securities and Exchange Commission.

AGTC to Host Conference Call on June 24 at 8:00 AM ET to Discuss Achromatopsia 12-Month Data in its Ongoing CNGB3 and CNGA3 Phase 1/2 Clinical Trials

Retrieved on: 
Wednesday, June 23, 2021

AGTC management will host a conference call and webcast with accompanying slides beginning at 8:00 AM Eastern Time on the same date to review the ACHM CNGB3 and CNGA3 data and provide an update on the trials.

Key Points: 
  • AGTC management will host a conference call and webcast with accompanying slides beginning at 8:00 AM Eastern Time on the same date to review the ACHM CNGB3 and CNGA3 data and provide an update on the trials.
  • The live webcast will be available in the Events and Presentations section of the Investor Relations page at http://ir.agtc.com/events-and-presentations .
  • AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3).
  • AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

AGTC to Present at American Optometric Association’s Optometry’s Meeting  

Retrieved on: 
Tuesday, June 22, 2021

Dr. Lin also will also lead three continuing education courses related to gene therapy and IRDs.

Key Points: 
  • Dr. Lin also will also lead three continuing education courses related to gene therapy and IRDs.
  • AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases.
  • AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3).
  • AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

KCSA Strategic Communications Expands Media Team with the Addition of Former CNBC Senior Producer Kristin Cwalinski

Retrieved on: 
Tuesday, June 22, 2021

NEW YORK, June 22, 2021 /PRNewswire/ -- KCSA Strategic Communications ("KCSA"), a leading New York City-based integrated communications firm, announced the hiring of Kristin Cwalinski as a Vice President on the Company's Public Relations team.Ms.

Key Points: 
  • NEW YORK, June 22, 2021 /PRNewswire/ -- KCSA Strategic Communications ("KCSA"), a leading New York City-based integrated communications firm, announced the hiring of Kristin Cwalinski as a Vice President on the Company's Public Relations team.Ms.
  • In her new position with KCSA, Ms. Cwalinski will serve as a senior media relations strategist and counselor.
  • She will utilize her expertise across the entire media landscape to assist in securing opportunities for the firm's clients.
  • "KCSA is focused on bringing in and growing the best talent in the communications industry, which is why we leapt at the opportunity to bring in Kristin.

AGTC Expands Leadership Team with Appointment of Key Regulatory Leader

Retrieved on: 
Wednesday, May 26, 2021

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases.

Key Points: 
  • AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases.
  • AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs.
  • AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3).
  • AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

Regulus Therapeutics Announces Incremental Update of Autosomal Dominant Polycystic Kidney Disease (ADPKD) Program

Retrieved on: 
Friday, May 21, 2021

Regulus believes these data demonstrate that RGLS4326 engages the target miR-17 leading to increased expression of the PKD1 and PKD2 genes and the resultant increases in polycystins\' levels.

Key Points: 
  • Regulus believes these data demonstrate that RGLS4326 engages the target miR-17 leading to increased expression of the PKD1 and PKD2 genes and the resultant increases in polycystins\' levels.
  • Regulus plans to discuss its approach to addressing the remaining partial clinical hold requirements with FDA in mid-2021.
  • These forward-looking statements are based upon Regulus\' current expectations and involve assumptions that may never materialize or may prove to be incorrect.
  • All forward-looking statements contained in this press release speak only as of the date on which they were made.

Saniona to Host Research and Development (R&D) Day on Leveraging Ion Channel Targeting Expertise for Rare Diseases

Retrieved on: 
Wednesday, May 12, 2021

Presenters will include members of Saniona\xe2\x80\x99s research team and executive management, as well as ion channel Key Opinion Leader (KOL) Heike Wulff, Ph.D., Professor of Pharmacology, University of California (UC) Davis School of Medicine.

Key Points: 
  • Presenters will include members of Saniona\xe2\x80\x99s research team and executive management, as well as ion channel Key Opinion Leader (KOL) Heike Wulff, Ph.D., Professor of Pharmacology, University of California (UC) Davis School of Medicine.
  • Dr. Wulff\xe2\x80\x99s research is focused on potassium channel pharmacology and the design of new ion channel modulating drugs and tool compounds.
  • Lead candidate SAN711 is entering Phase 1 for rare neuropathic disorders, with SAN903 for rare inflammatory and fibrotic disorders advancing through preclinical development.
  • Led by an experienced scientific and operational team, Saniona has an established research organization in Copenhagen, Denmark and is building its corporate office in the Boston, Massachusetts area, U.S.

AGTC to Host Third Quarter Financial Results Conference Call and Webcast on May 17, 2021

Retrieved on: 
Tuesday, May 11, 2021

AGTC management will host a conference call beginning at 4:30 PM Eastern Time on the same date to review results and provide a corporate update.\nTo access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US).

Key Points: 
  • AGTC management will host a conference call beginning at 4:30 PM Eastern Time on the same date to review results and provide a corporate update.\nTo access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US).
  • A live webcast will be available in the Events and Presentations section of the Investor Relations page at http://ir.agtc.com/events-and-presentations .
  • AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3).
  • AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

AGTC Reports Positive 12-Month Data from Highest Dose Groups in its Ongoing XLRP Phase 1/2 Clinical Trial

Retrieved on: 
Thursday, May 6, 2021

\xe2\x80\x9cGene therapy holds great promise in improving outcomes for these early and moderate staged XLRP patients, who today have no FDA-approved treatment options.

Key Points: 
  • \xe2\x80\x9cGene therapy holds great promise in improving outcomes for these early and moderate staged XLRP patients, who today have no FDA-approved treatment options.
  • \xe2\x80\x9cThe 50% response rate observed in Groups 5 and 6, coupled with the 12-month visual sensitivity and visual acuity data from these groups and 24-month data from three Group 4 patients, position the Phase 2/3 Vista trial and expanded Phase 1/2 Skyline trials for success.
  • AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3).
  • AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.