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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.


Solid Biosciences Announces Acquisition of AavantiBio and Concurrent $75 Million Private Placement

- Transactions to create a precision genetic medicine company focused on neuromuscular and cardiac rare diseases, led by industry veteran and current AavantiBio CEO, Bo Cumbo -

- Strong synergies expected by combining key assets, including product candidates for Duchenne muscular dystrophy, Friedreich’s ataxia, BAG3 mediated dilated cardiomyopathy and other undisclosed cardiac diseases, novel capsid libraries, and personnel -

- Combined company is expected to have approximately $215 million in cash and investments, which is expected to fund the combined company into 2025 and support attainment of key milestones for lead gene therapy programs -

- Companies to Host Conference Call today, September 30, 2022 at 8:00 AM ET -

CHARLESTOWN, Mass. and CAMBRIDGE, Mass., Sept. 30, 2022 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), and AavantiBio, Inc., a privately-held gene therapy company focused on transforming the lives of patients with Friedreich’s ataxia and rare cardiomyopathies, today announced that the companies have entered into a definitive merger agreement whereby Solid will acquire AavantiBio, including its pipeline assets and net cash. The combined company will focus on advancing a portfolio of neuromuscular and cardiac programs, led by SGT-003, a differentiated gene transfer candidate, for the treatment of Duchenne. Additional pipeline programs include AVB-202, a gene transfer candidate for the treatment of Friedreich’s ataxia, AVB-401 for BAG3 mediated dilated cardiomyopathy, and additional assets for the treatment of undisclosed cardiac diseases. Following approval by Solid stockholders, the combined company will operate as Solid Biosciences, will trade on Nasdaq under the ticker symbol “SLDB” and Bo Cumbo, the current Chief Executive Officer of AavantiBio, will assume the role of President and CEO of Solid Biosciences.

Read the Full Press Release here
 
 

Activation of a type I interferon response associated with acute frataxin knockdown in iPSC-derived cardiomyocytes

Frataxin functions in the biogenesis of iron-sulfur clusters, which are prosthetic groups that are found in proteins involved in many biological processes. To study the changes associated with decreased frataxin in human cardiomyocytes, the authors developed a novel isogenic model by acutely knocking down frataxin, post-differentiation, in cardiomyocytes derived from induced pluripotent stem cells. Transcriptome analysis of four biological replicates identified severe mitochondrial dysfunction and a type I interferon response as the pathways most affected by frataxin knockdown. In iPSC-derived cardiomyocytes, loss of frataxin leads to mitochondrial dysfunction. The type I interferon response was activated in multiple cell types following acute frataxin knockdown and was caused, at least in part, by release of mitochondrial DNA into the cytosol, activating the cGAS-STING sensor pathway.

Read the Full article here
 

Register before October 1st for discounted pricing for ICAR


The International Congress for Ataxia Research (ICAR) 2002 will be held November 1-4, 2022 in Dallas, TX and will include plenary sessions, poster sessions, breakouts and workshops. Featured topics include: gene discovery, disease mechanisms, cerebellar non-motor circuits and functions, emerging and existing therapeutics, biomarkers and late breaking research. ICAR will also feature workshops with practical demonstrations, discussions on challenges of new and established technologies, and sessions specifically designed by and for junior investigators. On October 1, the registration price will increase by $100.

Read More Here

Larimar Therapeutics Announces FDA has Partially Lifted the Clinical Hold and Plans for a Phase 2 Dose Exploration Trial of CTI-1601 in Friedreich’s Ataxia Patients

The FDA has cleared the initiation of 25 mg cohort of a Phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in Friedreich’s ataxia (FA) patients. In a written communication to Larimar, the FDA indicated it was lifting its full clinical hold on the CTI-1601 program and imposing a partial hold. The FDA will review data from the 25 mg cohort prior to escalating the dose in the second cohort. Larimar expects to begin the Phase 2 trial in Q4 2022.

Larimar’s upcoming Phase 2 trial is designed to further characterize CTI-1601’s safety, pharmacodynamic (PD), and pharmacokinetic (PK) profiles to provide information about the preferred long-term dose and dose regimen. Eligible patients will include ambulatory and non-ambulatory individuals with FA who are at least 18 years old.

Click here for the full press release which contains additional background and information on this program:

FARA is working with Larimar to host a webinar with Larimar Therapeutics for the patient community next week. We will share date and time and registration details in the next few days.
 
 

A promissing mouse model for Friedreich Ataxia progressing like human patients

Many groups worked on the creation of FRDA mouse models by decreasing the mouse frataxin or knocking it out, or by introducing a transgene with a human frataxin with long GAA repeat. Most of the mouse models are limited to one problem, either neurologic or cardiac symptoms, and, for those that have both, generally these symptoms are too severe, and mice have a very short life span, which does not reflect the human disease's progression. Jackson Laboratories Inc. developed a new mouse model that has 800 GAA repeats. The authors report here that the YG8-800 mice show progressively worsening locomotion and balance phenotypes starting at 11 to 20 weeks depending on the behavioral test. Moreover, these mice showed at 26 weeks a heart hypertrophy characterized by an increased heart to body weight ratio. YG8-800 mice are thus currently a promising mouse model for FRDA.

Read the Full article here
 

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