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Supported Research

The research listed below was partially supported by FARA thanks to your generosity.

For more information on FARA-funded research & scientists, please visit FARA Funded Research, Active Clinical Trials and the Featured Scientist.


Design Therapeutics Launches with $45 Million to Develop a New Class of Disease-Modifying Therapies for Serious Degenerative Disorders

Series A Financing Funds Novel Pipeline for Patients with Nucleotide Repeat Disorders

Company Advancing Lead Program for Friedreich’s Ataxia toward Clinical Development


San Diego, Calif., March 20, 2020 – Design Therapeutics announced today that it is launching to create and develop a new class of therapies for patients with serious degenerative disorders caused by nucleotide repeat expansions. The company has closed a $45 million Series A financing led by SR One, with participation from Cormorant Asset Management, Quan Capital and WestRiver Group, to advance its lead therapeutic candidate into clinical development for the treatment of Friedreich’s ataxia, and support advancement of its discovery programs for multiple other degenerative diseases, including fragile X syndrome and myotonic dystrophy. The company’s pipeline is led by a novel program for Friedreich’s ataxia. Design Therapeutics has developed a novel program that unblocks transcription, thereby restoring the natural production and function of frataxin. With the use of proceeds from the Series A fundraising, Design Therapeutic intends to conduct IND-enabling studies and initiate clinical development for its program for Friedreich’s ataxia.

Read the entire article HERE

Chondrial Therapeutics Announces Dosing of First Patients in Phase 1 Clinical Program of CTI-1601 for Treatment of Friedreich’s Ataxia

CTI-1601 granted Rare Pediatric Disease Designation and Fast Track Designation by U.S. FDA

BALA CYNWYD, Pa., Dec. 18, 2019 (GLOBE NEWSWIRE) -- Chondrial Therapeutics, a clinical-stage biotechnology company focused on developing treatments for rare diseases, with an initial focus on Friedreich’s ataxia (FA), today announced that the first patients have been dosed in a Phase 1 clinical trial to evaluate the safety and tolerability of single ascending doses of CTI-1601. CTI-1601 is a recombinant fusion protein intended to deliver human frataxin to patients with FA, who have decreased levels of frataxin.

"CTI-1601 is designed to address the root cause of Friedreich’s ataxia – low levels of frataxin. The initiation of our Phase 1 clinical program was supported by positive preclinical data and is an important step forward for patients affected by FA, a devastating and progressive disease for which there is no cure," said Carole Ben-Maimon, MD, President and Chief Executive Officer of Chondrial Therapeutics. "We separately announced today a merger that will provide us with significant resources to advance our development of CTI-1601 and expand our efforts in other rare diseases that may benefit from a cell-penetrating peptide approach."

View the entire Press Release Here

Exicure Announces First Neurological Development Program in Friedreich’s Ataxia and Expands Scientific Advisory Board

- Exicure to collaborate with Friedreich’s Ataxia Research Alliance (FARA)

- Hank Paulson, MD, PhD and Susan Perlman, MD, added to Scientific Advisory Board


CHICAGO & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dec. 16, 2019-- Exicure, Inc. (NASDAQ:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) technology, today announced Friedreich’s ataxia (FA) as the therapeutic indication for the company’s first neurology development program. Exicure also announced the expansion of its Scientific Advisory Board.

"Over the past year Exicure has developed extensive preclinical data supporting the development of our SNAs for neurological disorders. We’re eager to evaluate our technology for this important unmet medical need," said David Giljohann, PhD, Exicure’s chief executive officer. "We are also pleased to add the experience and team from the Friedreich’s Ataxia Research Alliance (FARA)," added Dr. Giljohann.


View the Exicure Press Release PDF Here

Reata Announces Positive Topline Results from the MOXIe Registrational Trial of Omaveloxolone in Patients with Friedreich’s Ataxia

ACHIEVED PRIMARY ENDPOINT OF STATISTICALLY SIGNIFICANT IMPROVEMENT IN MFARS COMPARED TO PLACEBO AFTER 48 WEEKS OF TREATMENT

CONFERENCE CALL WITH MANAGEMENT SCHEDULED FOR OCTOBER 15, 2019, AT 8:00 AM ET

IRVING, Texas, Oct. 14, 2019 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (Nasdaq: RETA), a clinical-stage biopharmaceutical company, announced today that the registrational Part 2 portion of the MOXIe Phase 2 trial of omaveloxolone in patients with Friedreich’s ataxia (FA) met its primary endpoint of change in the modified Friedreich’s Ataxia Rating Scale (mFARS) relative to placebo after 48 weeks of treatment. Patients treated with omaveloxolone (150 mg/day) demonstrated a statistically significant, placebo-corrected 2.40 point improvement in mFARS after 48 weeks of treatment (p=0.014). Omaveloxolone treatment was generally reported to be well-tolerated. Based on these positive results, and subject to discussions with regulatory authorities, the company plans to proceed with the submission of regulatory filings for marketing approval in the United States and internationally.

Read the entire article HERE

Pharmacokinetics and pharmacodynamics of the novel Nrf2 activator omaveloxolone in primates

Omaveloxolone is a potential therapy thatactivates Nrf2, a master transcription factor that regulates genes with antioxidative, anti-inflammatory, and mitochondrial bioenergetic properties, and is being evaluated in patients with Friedreich's ataxia. This study evaluated the pharmacokinetics (PK), pharmacodynamics (PD) and tissue distribution of omaveloxolone in monkeys after single and multiple oral doses, and then compared these data to initial results in Friedreich's ataxia patients. A PK/PD model was generated with the monkey data, and used to further evaluate the Friedreich's ataxia patient PK profile. The authors found that oral administration of omaveloxolone to monkeys was associated with dose-linear plasma PK and readily measurable and dose-proportional concentrations in liver, lung, and brain. Dose-dependent induction of Nrf2 target genes was also observed. Clinically, oral administration of omaveloxolone to Friedreich's ataxia patients at incremental doses from 2.5 to 300 mg produced dose-proportional systemic exposures. Clinical doses of at least 80 mg were associated with meaningful improvements in neurological function in patients and generated plasma omaveloxolone concentrations consistent with those significantly inducing Nrf2 target genes in monkeys, as shown with the monkey PK/PD model. Overall, the monkey data demonstrate a well-characterized and dose-proportional PK and tissue distribution profile after oral administration of omaveloxolone, which was associated with Nrf2 activation. Further, systemic exposures to omaveloxolone that produce Nrf2 activation in monkeys were readily achievable in Friedreich's ataxia patients after oral administration.

Read the entire article HERE

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