Eric Sorscher

ReCode Therapeutics Presents Preclinical Data Using SORT-LNP™ and RNA Platforms to Rescue CFTR Function at the 44th European Cystic Fibrosis Conference (ECFS)

Friday, June 11, 2021 - 1:00pm

These data showcase the potential of our differentiated RNA-based molecular therapies to treat cystic fibrosis.

Key Points: 
  • These data showcase the potential of our differentiated RNA-based molecular therapies to treat cystic fibrosis.
  • By leveraging our LNP platform to deliver genomic medicines, we have demonstrated the ability to restore cystic fibrosis transmembrane conductance regulator (CFTR) function through both tRNA and mRNA approaches, said David Lockhart, Ph.D., CEO & President, ReCode Therapeutics.
  • RCT223 was shown to restore CFTR function for at least 72 hours in patient-derived hBE cells after a single administration.
  • The Companys pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia.

Metagenomi Receives Award From Cystic Fibrosis Foundation to Explore Novel Gene Editing Systems for Therapeutic Applications for Cystic Fibrosis

Thursday, May 27, 2021 - 1:30pm

Metagenomi , a gene editing company, today announced that it has received an award from the Cystic Fibrosis Foundation to identify and evaluate novel gene editing systems for the development of therapeutics to treat cystic fibrosis.

Key Points: 
  • Metagenomi , a gene editing company, today announced that it has received an award from the Cystic Fibrosis Foundation to identify and evaluate novel gene editing systems for the development of therapeutics to treat cystic fibrosis.
  • Metagenomis gene editing capabilities are transformational and could provide opportunities for gene editing-based therapeutics that are not possible with other systems.
  • We are committed to leveraging our advanced gene editing toolbox for therapeutic applications across disease areas, including cystic fibrosis, and we look forward to collaborating with the Cystic Fibrosis Foundation to broaden therapeutic options for patients.
  • In cystic fibrosis patients, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional.

Translate Bio Highlights mRNA Platform Potential for the Treatment of Cystic Fibrosis (CF) at the 34th Annual North American Cystic Fibrosis Conference

Wednesday, October 7, 2020 - 9:05pm

The presentations also highlight the potential benefits of a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) mRNA which may include lower dosing requirements and/or reduced administration time.

Key Points: 
  • The presentations also highlight the potential benefits of a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) mRNA which may include lower dosing requirements and/or reduced administration time.
  • Preclinical studies are ongoing to select the optimal mRNA sequence and lipid nanoparticle (LNP) for Translate Bios next generation CFTR mRNA product.
  • Translate Bios lead pulmonary candidate is being evaluated as an inhaled treatment for cystic fibrosis (CF) in a Phase 1/2 clinical trial.
  • Translate Bio is also pursuing the development of mRNA vaccines for infectious diseases under a collaboration with Sanofi Pasteur.

Calithera Biosciences’ CB-280 Arginase Inhibitor Trial in Progress Poster Presented at the North American Cystic Fibrosis 2020 Virtual Conference

Wednesday, October 7, 2020 - 1:08pm

The randomized, double-blind, placebo-controlled, multiple ascending dose-escalation study (NCT04279769) is exploring CB-280 versus placebo in adults with cystic fibrosis and chronic infection with Pseudomonas aeruginosa who are stable on cystic fibrosis medications, including cystic fibrosis transmembrane conductance regulator (CFTR) modulators.

Key Points: 
  • The randomized, double-blind, placebo-controlled, multiple ascending dose-escalation study (NCT04279769) is exploring CB-280 versus placebo in adults with cystic fibrosis and chronic infection with Pseudomonas aeruginosa who are stable on cystic fibrosis medications, including cystic fibrosis transmembrane conductance regulator (CFTR) modulators.
  • Enrollment in this study is ongoing and Calithera expects to share interim data in 2021.
  • The poster presentation includes preclinical study results which suggest CB-280 significantly improved lung function and reduced Pseudomonas aeruginosa colony-forming units in pre-clinical models.
  • CB-280 is the first arginase inhibitor to be evaluated for the treatment of cystic fibrosis, said Susan Molineaux, PhD, president and chief executive officer of Calithera.

New FDA-approved cystic fibrosis medicine, Trikafta®, among other specialty drugs available through AllianceRx Walgreens Prime

Monday, January 6, 2020 - 3:00pm

It is caused by a defective protein that results from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Key Points: 
  • It is caused by a defective protein that results from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
  • "We are thrilled to be able to offer this new therapy," says Tracey James, RPh, senior vice president of pharmacy services at AllianceRx Walgreens Prime.
  • Trikafta is a specialty medicine primarily distributed through a limited network of specialty pharmacies and distributors, which includes AllianceRx Walgreens Prime.
  • AllianceRx Walgreens Prime ( alliancerxwp.com ) is a specialty and home delivery pharmacy that strives to provide exceptional care throughout a patient's treatment journey with the medications they need every day.

Corbus Pharmaceuticals Completes Enrollment of Phase 2b Study of Lenabasum for Treatment of Cystic Fibrosis

Monday, November 18, 2019 - 1:17pm

Corbus has enrolled 426 individuals with CF in the Phase 2b international, multicenter, randomized, double-blind, placebo-controlled study that is being conducted in North America, Europe, and Israel.

Key Points: 
  • Corbus has enrolled 426 individuals with CF in the Phase 2b international, multicenter, randomized, double-blind, placebo-controlled study that is being conducted in North America, Europe, and Israel.
  • The Phase 2b CF study is funded in part by a Development Award for up to $25 Million from the Cystic Fibrosis Foundation.
  • Lenabasum treatment also was associated with a lower rate of and longer time to pulmonary exacerbations in a Phase 2 cystic fibrosis study.
  • Cystic fibrosis (CF) is a chronic, rare, genetic disease caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

AllianceRx Walgreens Prime study demonstrates favorable adherence, but high cost for cystic fibrosis patients prescribed modulator medications

Wednesday, November 6, 2019 - 2:00pm

ORLANDO, Fla., Nov. 6, 2019 /PRNewswire/ --AllianceRx Walgreens Prime and Duquesne University School of Pharmacy recently announced results from research analyzing data from cystic fibrosis (CF) patients prescribed cystic fibrosis transmembrane conductance regulator(CFTR) modulator medications.

Key Points: 
  • ORLANDO, Fla., Nov. 6, 2019 /PRNewswire/ --AllianceRx Walgreens Prime and Duquesne University School of Pharmacy recently announced results from research analyzing data from cystic fibrosis (CF) patients prescribed cystic fibrosis transmembrane conductance regulator(CFTR) modulator medications.
  • Researchers wanted to understand if patients took their medications as prescribed, as well as the financial impact of the medications on patients.
  • Since the introduction of the first CFTR modulator in 2012 and subsequent CFTR modulator approvals, these medications have significantly changed the treatment of CF.
  • Of the 3,482 patients using CFTR modulator therapies, 50.8% of patients were on lumacaftor/ivacaftor followed by tezacaftor/ivacaftor (24.8%) and ivacaftor (24.4%).

Prime Therapeutics forecasts cystic fibrosis spending could double in 2019

Tuesday, March 26, 2019 - 1:00pm

EAGAN, Minn., March 26, 2019 /PRNewswire/ -- Recently approved cystic fibrosis (CF) treatments and new approvals for additional ages or mutations could double the spending on CF treatments in 2019, according to new research by Prime Therapeutics LLC (Prime), a pharmacy benefit manager (PBM) serving 23 Blue Cross and Blue Shield plans nationally.

Key Points: 
  • EAGAN, Minn., March 26, 2019 /PRNewswire/ -- Recently approved cystic fibrosis (CF) treatments and new approvals for additional ages or mutations could double the spending on CF treatments in 2019, according to new research by Prime Therapeutics LLC (Prime), a pharmacy benefit manager (PBM) serving 23 Blue Cross and Blue Shield plans nationally.
  • Three treatments called cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies have been approved by the U.S. Food and Drug Administration (FDA).
  • FDA approvals broadening CFTR modulator use and a triple combination product are anticipated by the end of 2019.
  • Prime Therapeutics LLC (Prime) helps people get the medicine they need to feel better and live well.