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Scientific News

FARA funds research progress

In this section, you will find the most recent FA research publications, many of which are funded by FARA, as well as information on upcoming conferences and symposiums. You can search for articles by date using the archive box in the right hand column. To locate FARA Funded or Supported Research, click the hyperlink in the right hand column. You may also search for specific content using key words or phrases in the search button at the top right of your screen. Please be sure to visit other key research sections of our website for information on FARA’s Grant Program and the Treatment Pipeline.



Reata Announces First Patient Enrolled in Part 2 of MOXIe Study of Omaveloxolone for the Treatment of Friedreich’s Ataxia

Reata Pharmaceuticals today announced the enrollment of the first patient in the pivotal Part 2 of the MOXIe trial to evaluate omaveloxolone in patients with Friedreich’s ataxia (FA).

"Friedreich’s ataxia is a severe, neurological disorder that profoundly impacts patients and their families. Based on the results from Part 1 of MOXIe, we are optimistic that Part 2 of the MOXIe trial could position omaveloxolone to become the first therapy approved for patients with Friedreich’s ataxia," said Warren Huff, Reata's Chief Executive Officer and President. "With the initiation of Part 2 of MOXIe, Reata has launched three pivotal programs in the last 12 months."

Read the entire Press Release HERE

Friedreich Ataxia: Developmental Failure of the Dorsal Root Entry Zone

Dorsal root ganglia, dorsal roots (DR), and dorsal root entry zones (DREZ) are vulnerable to frataxin deficiency in Friedreich ataxia (FA). A previously unrecognized abnormality is the intrusion of astroglial tissue into DR. Segments of upper lumbar spinal cord of 13 homozygous and 2 compound heterozygous FA patients were studied to look at structures in the DREZ. Normal DREZ showed short, sharply demarcated, dome-like extensions of CNS tissue into DR. The Schwann cell-related proteins formed tight caps around these domes. In FA, CNS tissue extended across DREZ and into DR over much longer distances by breaching the CNS-PNS barrier. The transition between PNS and CNS myelin proteins was disorganized. During development, neural-crest derived boundary cap cells provide guidance to dorsal root ganglia axons growing into the dorsal spinal cord and at the same time block the inappropriate intrusion of CNS glia into DR. It is likely that frataxin is required during a critical period of permissive (axons) and nonpermissive (astroglia) border-control.

Read the entire article HERE

Sustained FXN expression in dorsal root ganglia from a nonreplicative genomic HSV-1 vector

With the aim of developing a gene therapy for FA neuropathology,the authors describe describe the construction and preliminary characterization of a high capacity nonreplicative genomic herpes simplex virus type 1 vector (H24B-FXNlac vector) carrying a reduced version of the human FXN genomic locus, comprising the 5 kb promoter and the FXN cDNA with the inclusion of intron 1. They show that the transgene cassette contains the elements necessary to preserve physiological neuronal regulation of human FXN expression. Transduction of cultured fetal rat dorsal root ganglion neurons with the H24B-FXNlac vector results in sustained expression of human FXN transcripts and frataxin protein. Rat footpad inoculation with the H24B-FXNlac vector results in human FXN transgene delivery to the dorsal root ganglia, with expression persisting for at least 1 month. They conclude that theirr results support the feasibility of using this vector for sustained neuronal expression of human frataxin for FA gene therapy.

Read the entire article HERE

BioMarin Highlights Breadth of Innovative Development Pipeline at R&D Day on Oct 18th in New York

SAN RAFAEL, Calif., Oct. 18, 2017 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) updated the investment community on the Company's development portfolio, which is focused on innovative therapies to treat rare and ultra-rare diseases.

"We are pleased to share the progress of our development programs in therapies to treat rare genetic diseases; hemophilia A, PKU, achondroplasia and our next IND into Friedreich's Ataxia," said Hank Fuchs, M.D., President Worldwide Research and Development of BioMarin. "In the near term, we are expecting an FDA decision on pegvaliase to treat adults with uncontrolled PKU in the first half of next year, and we continue to be rapidly and decisively developing the potential first gene therapy for severe hemophilia A."

Read the entire article HERE

CRISPR Therapeutics Awarded Grant from FARA to Collaborate with University of Alabama at Birmingham on Gene-edited Treatments for Friedreich’s Ataxia

ZUG, Switzerland and CAMBRIDGE, Mass., Oct. 13, 2017 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP), a genome editing company focused on creating transformative medicine for serious diseases, today announced the receipt of the Kyle Bryant Translational Research Award from Friedreich’s Ataxia Research Alliance (FARA), a non-profit organization that is focused on curing Friedreich’s Ataxia (FA). The grant is awarded to fund research on in vivo CRISPR/Cas9-based gene-editing approaches to treat FA, which will be performed in collaboration with Dr. Marek Napierala at University of Alabama at Birmingham. This announcement coincides with FARA’s rideATAXIA Philadelphia event, a lead location in an annual bike ride program founded by patient Kyle Bryant, that increases FA awareness and raises funds to treat and cure FA through research.

Read the full Press Release HERE

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