Author Archives: Margarida Azevedo, MSc

Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…

Chondrial Therapeutics Licenses Friedreich’s Ataxia Therapy, Plans Further Development

Chondrial Therapeutics has licensed the investigational compound CTI-1601 for treating  Friedreich’s ataxia (FA) from Indiana University Research and Technology Corporation (IURTC) and Wake Forest University Health Sciences. The company said it has also secured $22.6 million in financing to advance the treatment. “The $22.6 million Series A financing, combined with the licensing…

Friedreich’s Ataxia Symposium Oct. 17 Marks Increase in Specialized Care, Community Outreach

The Friedreich’s Ataxia Center of Excellence at The Children’s Hospital of Philadelphia is presenting a one-day annual symposium on Friedreich’s ataxia (FA) Monday, Oct. 17, in King of Prussia, Pennsylvania. The ninth annual meeting demonstrates the ongoing outreach of the Friedreich’s Ataxia Research Alliance (FARA) to the FA community nationwide and an…

Potential Friedreich’s Ataxia Treatment, RT001, Shows Safety and Tolerability in Clinical Trial

Early results from Retrotope‘s Phase 1/2 clinical trial assessing RT001 as a potential treatment for Friedreich’s ataxia (FA) show that it has met it primary safety, tolerability and pharmacokinetics endpoints, the study’s lead investigator announced. The ongoing trial, titled “A First in Human Study of RT001 in Patients with Friedreich’s Ataxia” (NCT02445794),…

Stretching Aid Meant to Prevent Injuries, Flexfit, Seen to Benefit People with Chronic Ills

KRAM Wellness’ flagship product, Flexfit, is a three-in-one injury prevention product that provides a controlled stretch using an adjustable, non-elastic design. The company reports that, in addition to aiding fitness fans, this newly available device is used by chiropractors and physical therapists, and may be of benefit to people with dexterity problems, like Friedreich’s…

Global Genes Helps Hundreds Attend Annual RARE Patient Advocacy Summit by Awarding $100,000 in Travel Stipends

The nonprofit Global Genes, an organization for patients and families fighting rare and genetic diseases, is awarding more than $100,000 in travel stipends to help hundreds of patient advocates attend 5th Annual RARE Patient Advocacy Summit  Sept. 22-23 in Huntington Beach, California. After the summit, the 2016 Tribute to Champions of…

Pfizer Purchases Bamboo Therapeutics, Heightens Position in Gene Therapy Industry

Pfizer recently acquired the biotech Bamboo Therapeutics which develops therapies for potential treatment of patients with neuromuscular and central nervous system (CNS) rare diseases – like Friedreich’s ataxia (FA). The purchase is expected to expand Pfizer’s portfolio in gene therapy by including the advanced recombinant Adeno-Associated Virus (rAAV) vector design and production technology; and a fully…

New Kind of Sunscreen Targeting Free Iron in Cells May Also Treat Friedrich’s Ataxia

A new form of sun protection that works by chelating, or binding, excess iron within mitochondria may not only protect against UVA rays that current lotions are unable to block, but also lead to new treatments for  Friedreich’s ataxia (FA). The compound, named “mitoiron claw” by the scientific team in the U.K. that developed it, is thought capable…

California NAACP Joins Fridreich’s Ataxia Research Alliance, Other Groups to Discuss Controversial Healthcare Bill

The Friedreich’s Ataxia Research Alliance (FARA) joined the California NAACP and other patient advocacy groups to listen to opinions and concerns about SB 1010 — a controversial California state bill that could potentially impact Friedreich’s ataxia (FA) care in the state. Those opposed to the bill, sponsored by state Sen. Ed Hernandez, claim it “prioritizes…

New Partnership Working to Develop and Advance a Gene Therapy for Friedreich’s Ataxia

Agilis Biotherapeutics, LLC, and Waisman Biomanufacturing have signed an exclusive partnership agreement to develop and produce AGIL-FA, Agilis’ potential gene therapy for the treatment of Friedreich’s ataxia (FA). Agilis, a biotechnology company specializing in therapies for rare genetic diseases that affect the central nervous system (CNS), and Waisman, a nonprofit gene…

Promising Friedreich’s Ataxia Therapy Seen in Synthetic RNA and DNA Agents Used by UT Researchers

University of Texas (UT) Southwestern Medical Center researchers have identified RNA and DNA synthetic agents that increase frataxin levels and alleviate Friedreich’s ataxia (FA), suggesting that synthetic nucleic acids that target the specific disease-causing mutation can be lead compounds for FA therapy. The research paper, “Activating frataxin expression by repeat-targeted nucleic acids,”…

Friedreich’s Ataxia Clinical Study of Oral Drug, RT001, Enrolling New Cohort of Patients

Retrotope announced the completion of a first dosing cohort, and the opening of patient enrollment for a highest dose cohort, in its ongoing, 28-day clinical study of orally dosed RT001 in Friedreich’s ataxia (FA) patients. The treatment to date has been well-tolerated, with no serious adverse effects or dose limiting toxicities, the pharmaceutical company reported. “We…

In Friedreich’s Ataxia, Neurotrophins Hold Therapeutic Potential to Prevent, Treat Neurodegeneration

Researchers have demonstrated in the lab (in vitro) and in living subjects (in vivo), that gene transfer of brain-derived neurotrophic factor (BDNF) protected models of Friedreich’s ataxia from neuronal degenerative death, providing a proof of principle that neurotrophins might be a therapeutic option to prevent and treat neurodegeneration associated with Friedreich’s ataxia.

Friedreich’s Ataxia Drug Developer RaNA Therapeutics to Present Data at Conference

RaNA Therapeutics, a Cambridge, Massachusetts-based biotech company that develops next-generation RNA-targeted medicines and which currently has a lead program in Friedreich’s Ataxia, announced that its CEO Ronald Renaud will present a corporate overview on Feb. 8 at the 18th annual BIO CEO & Investor Conference in New York City. RaNA’s management will also…

Avalanche to Expand Gene Therapy Holdings by Acquiring Annapurna

Avalanche Biotechnologies, Inc., announced that it has entered into a “definitive agreement” to acquire the French company Annapurna Therapeutics SAS. The merger, expected to close by mid-2016, has been approved by the boards of directors of both companies but is subject to approval by Avalanche stockholders. Under the agreement’s terms, Avalanche will acquire the totality…

Promising Friedrich’s Ataxia Drug Candidate Gains Support of NIH Scientists

Chondrial Therapeutics recently announced that further development of its lead drug candidate for the treatment of Friedreich’s ataxia, TAT-Frataxin (TAT-FXN), will be aided by researchers with the Therapeutics for Rare and Neglected Diseases (TRND) program, run through the National Institutes of Health (NIH), with the aim of filing an Investigational New Drug (IND) application for the therapy and…

Potential Friedreich’s Ataxia Therapy Advances with FARA’s Support

Catabasis Pharmaceuticals, Inc., recently announced that it will receive the Kyle Bryant Translational Research Award, given by the Friedreich’s Ataxia Research Alliance (FARA), to further evaluate CAT-4001, a potential Friedreich’s ataxia (FA) drug. CAT-4001 is being developed by Catabasis as a potential treatment for neurodegenerative diseases, such as FA and amyotrophic lateral…

Annapurna Therapeutics Partners with Weill Cornell Medicine to Advance Gene Therapy

Annapurna Therapeutics has just announced it will collaborate with Weill Cornell Medicine to develop a market-leading partnership specialized in gene therapy. One of these therapies currently under Annapurna’s exclusive development targets cardiomyopathy as it is associated with Friedreich’s ataxia, a disease that affects between 10,000 and 20,000 people in the U.S. and Europe. Friedreich’s ataxia…

Retrotope Announces Second Clinical Trial Site For Friedreich’s Ataxia Study Open for Enrollment

Retrotope recently announced a second clinical trial site, the Collaborative Neuroscience Network, LLC (CNS), has opened in Long Beach, California, as part of the company’s ongoing 28-day, first-in-human randomized, double-blind, controlled, ascending dose study of orally dosed experimental therapy RT001. The study will evaluate the safety, tolerability, pharmacokinetics (PK), disease state and exploratory endpoints in…

USF and FARA to Co-Host Friedreich’s Ataxia Symposium

A symposium focused on the rare genetic disease Friedreich’s ataxia, titled, “Understanding Energy for A Cure“, will gather some of the most prominent researchers and disease experts under one roof and help bridge the communication gap between patients and reliable sources of information and updates on FA. The event is hosted by the University…

MDA and FARA Form Research Collaboration for Friedreich’s Ataxia Treatments

The Muscular Dystrophy Association (MDA), together with the Friedreich’s Ataxia Research Alliance (FARA), have announced a new strategic partnership geared towards discovering better ways to address Friedreich’s Ataxia (FA) through advancing research, therapeutic development and clinical care. This alliance further strengthens the former’s dedication to forming partnerships both with for- and not-for-profit…

Muscular Dystrophy Association to Fund 250 Research Projects with Grants Worth $10 Million

The Muscular Dystrophy Association, the world’s leading nonprofit health agency dedicated to save and improve the lives of people with muscle disease amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases, recently announced it awarded some of the world’s most promising and esteemed scientists research grants amounting to $10 million to fund…

Retrotope Opens Patient Enrolment in Florida to Test Friedreich’s Ataxia Drug

Retrope, Inc., a privately held clinical-stage pharmaceutical company leading the advance of a revolutionary new unifying theory of aging and degeneration, has just opened patient enrolment for a 28-day, first-in-human, randomized, double-blind, controlled, ascending dose study of oral drug RT001. The study aims to determine the drug’s safety, tolerability and pharmacokinetic profile,…

Novel Gene Therapy for Friedreich’s Ataxia by AAVLife Granted US Patent

Gene-therapy specialist company, AAVLife, recently acquired a patent from the United States Patent and Trademark Office for an experimental treatment of cardiomyopathy caused by the genetic disorder, Friedreich’s ataxia, which affects the nervous system and causes movement problems. People with this condition develop impaired muscle coordination (ataxia) that worsens over time and commonly…

Horizon Pharma Begins Phase 3 Trial of ACTIMMUNE For The Treatment of Patients With Friedreich’s Ataxia

Horizon Pharma has recently announced it initiated a Phase 3 study evaluating the Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich’s Ataxia study (“STEADFAST”) of ACTIMMUNE (interferon gamma-1b) for the treatment of people with Friedreich’s Ataxia (FA). ACTIMMUNE (Interferon gamma 1-b) is a biologically manufactured protein similar to…

Friedreich’s Ataxia Research Alliance Enters Venture Network

The Research Acceleration and Innovation Network (TRAIN) has invited nonprofit organization Friedreich’s Ataxia Research Alliance (FARA), which works nationwide to support research and find a cure for the rare neuromuscular disease Friedreich’s ataxia (FA), to join its network of organizations. The program was launched in 2005 by FasterCures and…

Horizon’s FA Therapy Granted FDA Fast Track Designation

Specialty biopharmaceutical Horizon Pharma plc has been awarded Fast Track status from the U.S. Food and Drug Administration (FDA) for the company’s ACTIMMUNE (interferon gamma-1b) to be used in patients with Friedreich’s ataxia (FA). ACTIMMUNE is a biologically developed protein resembling one produced naturally by the body to prevent infection,…

Intrexon Makes Offer to Acquire Exemplar Genetics

Intrexon Corporation has made an offer for the total acquisition of Exemplar Genetics, which is a company dedicated to study the treatment of life-threatening conditions, such as Friedreich’s ataxia, heart disease, cancer, cystic fibrosis, cardiac arrhythmia, as well as neuromuscular and neurodegenerative disorders. The company provides transgenic…