Genes

FSHD Society offers CME-accredited masterclass on FSH muscular dystrophy

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Wednesday, July 14, 2021

RANDOLPH, Mass., July 14, 2021 /PRNewswire-PRWeb/ --The FSHD Society is offering its first-ever CME-accredited masterclass on facioscapulohumeral muscular dystrophy (FSHD) .

Key Points: 
  • RANDOLPH, Mass., July 14, 2021 /PRNewswire-PRWeb/ --The FSHD Society is offering its first-ever CME-accredited masterclass on facioscapulohumeral muscular dystrophy (FSHD) .
  • Although FSHD is one of the most common forms of muscular dystrophy, it is often under-diagnosed, and diagnosed patients don't always benefit from the existing standards of care.
  • The FSHD Society is the world's largest research-focused patient advocacy organization for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy.
  • The FSHD Society has transformed the landscape for FSHD research and is committed to making sure that no one faces this disease alone.

Dyne Therapeutics Presents Preclinical Data from its Facioscapulohumeral Muscular Dystrophy Program During the FSHD Society International Research Congress

Retrieved on: 
Friday, June 25, 2021

(Nasdaq: DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, is presenting today preclinical data from its facioscapulohumeral muscular dystrophy (FSHD) program during the 28 th Annual FSHD Society International Research Congress .

Key Points: 
  • (Nasdaq: DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, is presenting today preclinical data from its facioscapulohumeral muscular dystrophy (FSHD) program during the 28 th Annual FSHD Society International Research Congress .
  • Dynes approach is designed to address the genetic basis of the disease by reducing DUX4 mRNA expression.
  • The companys lead FSHD candidate consists of a transferrin 1 receptor-binding fragment antibody (Fab) conjugated to a phosphorodiamidate morpholino oligomer (PMO) targeting DUX4 mRNA.
  • We are excited to share these data, which build upon our prior in vitro studies and support our approach to targeting the genetic basis of FSHD.

Surrozen Presents Data at 2021 International Liver Conference for Wnt-Modulating Antibody SZN-043

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Friday, June 25, 2021

The one oral presentation and three poster presentations discussed data from studies of SZN-043, Surrozens hepatocyte-targeted R-spondin mimetic, in mice and various disease models.

Key Points: 
  • The one oral presentation and three poster presentations discussed data from studies of SZN-043, Surrozens hepatocyte-targeted R-spondin mimetic, in mice and various disease models.
  • SZN-043 showed target-mediated drug disposition and pharmacokinetics that were consistent with that of an IgG-based molecule binding to the hepatocyte membrane protein ASGR1.
  • Surrozen is a biotechnology company discovering and developing drug candidates to selectively modulate the Wnt pathway.
  • Investors and security holders of Surrozen are advised to read, when available, the proxy statement/prospectus in connection with the written consent of Surrozen stockholders.

Arrowhead Presents Preclinical Data on ARO-DUX4 at FSHD Society International Research Congress

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Friday, June 25, 2021

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today presented preclinical data on the development of ARO-DUX4, the companys investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with facioscapulohumeral muscular dystrophy (FSHD), at the 28th Annual FSHD Society International Research Congress.

Key Points: 
  • Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today presented preclinical data on the development of ARO-DUX4, the companys investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with facioscapulohumeral muscular dystrophy (FSHD), at the 28th Annual FSHD Society International Research Congress.
  • Importantly, in a transgenic mouse model of FSHD, treatment with ARO-DUX4 prevented and reversed tamoxifen-induced increases in DUX4 and DUX4 target gene expression.
  • Collectively, these preclinical results suggest that ARO-DUX4 achieved good target engagement, which led to a downstream beneficial physiological change.
  • Preclinical data demonstrated that Arrowheads Targeted RNAi Molecule (TRiM) muscle delivery platform can achieve functional delivery to various types of skeletal muscle.

Fulcrum Therapeutics Announces Multiple Presentations at the 28th Annual FSHD Society International Research Congress (IRC)

Retrieved on: 
Monday, June 21, 2021

FSHD is a serious, rare, progressive and disabling disease for which there are no approved treatments.

Key Points: 
  • FSHD is a serious, rare, progressive and disabling disease for which there are no approved treatments.
  • FSHD is characterized by muscle degeneration and fat infiltration, initially affecting movement of the face and eventually the arms, trunk and legs.
  • Disease progression results in accumulation of disability, with many patients ultimately becoming dependent upon the use of a wheelchair for daily mobility.
  • FSHD is caused by mis-expression of DUX4 in skeletal muscle, resulting in the presence of DUX4 proteins that are toxic to muscle tissue.

PRR Announces Agency Owners Assuming Managing Principal Roles

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Tuesday, May 25, 2021

SEATTLE, May 25, 2021 /PRNewswire/ -- PRR, a women-owned and employee-owned, integrated communications agency, announced today that co-owners Malika Klingler and Diana Steeble are stepping into the role of managing principals to oversee the strategic direction of the agency.

Key Points: 
  • SEATTLE, May 25, 2021 /PRNewswire/ -- PRR, a women-owned and employee-owned, integrated communications agency, announced today that co-owners Malika Klingler and Diana Steeble are stepping into the role of managing principals to oversee the strategic direction of the agency.
  • During her tenure, Foster led the company to financial success and was integral in positioning PRR as an anti-racism agency.
  • "We are excited that Diana and Malika will bring their collective skills and talents to continue in leadership roles that best serve PRR today and into the future," said Colleen Gants, co-president of PRR.
  • Established in 1981, PRR is a mission driven agency dedicated to creating real and lasting change in the world.

Fulcrum Therapeutics Announces U.S. Food and Drug Administration Grants Fast Track Designation to Losmapimod for the Potential Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Retrieved on: 
Wednesday, May 12, 2021

Utilizing its internal product engine, Fulcrum discovered that inhibition of p38\xce\xb1/\xce\xb2 reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD.

Key Points: 
  • Utilizing its internal product engine, Fulcrum discovered that inhibition of p38\xce\xb1/\xce\xb2 reduced expression of the DUX4 gene in muscle cells derived from patients with FSHD.
  • Fulcrum\xe2\x80\x99s proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression.
  • The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
  • However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.\n'

Arrowhead Announces ARO-DUX4 as First Muscle Targeted RNAi Candidate Using TRiMTM Platform

Retrieved on: 
Thursday, April 15, 2021

b'Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced ARO-DUX4 as Arrowhead\xe2\x80\x99s first muscle targeted investigational RNAi therapeutic candidate to utilize its proprietary Targeted RNAi Molecule (TRIMTM) platform.

Key Points: 
  • b'Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced ARO-DUX4 as Arrowhead\xe2\x80\x99s first muscle targeted investigational RNAi therapeutic candidate to utilize its proprietary Targeted RNAi Molecule (TRIMTM) platform.
  • Pending abstract acceptance, Arrowhead intends to present preclinical data on ARO-DUX4 at the 28th Annual FSHD Society International Research Congress being held virtually on June 24-25, 2021.
  • As DUX4 expression is recognized as the cause of muscle pathology in FSHD patients, Arrowhead believes that the selective targeting and knockdown of DUX4 using RNAi may prevent or reverse downstream myotoxicity and lead to muscle repair and improvement in muscle function in patients.
  • Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes.

BioTheryX Announces Expansion of Scientific Advisory Board

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Wednesday, April 7, 2021

SAN DIEGO, April 7, 2021 /PRNewswire/ --BioTheryX, Inc., a clinical-stage company focused on degrading proteins to create life-saving medicines, today announced the expansion of its Scientific Advisory Board (SAB) with the appointment of three new members: David Komander, Ph.D., Kevan M. Shokat, Ph.D. and Peter Walter, Ph.D.

Key Points: 
  • SAN DIEGO, April 7, 2021 /PRNewswire/ --BioTheryX, Inc., a clinical-stage company focused on degrading proteins to create life-saving medicines, today announced the expansion of its Scientific Advisory Board (SAB) with the appointment of three new members: David Komander, Ph.D., Kevan M. Shokat, Ph.D. and Peter Walter, Ph.D.
  • His research work focuses on signaling pathways regulating innate immunity and inflammation, particularly in the context of cancer.
  • His lab deciphered key steps in the activation of the NFkB and Wnt signaling pathways and studied them in animal models of cancer.
  • This technology is designed to utilize the body's own protein disposal system to selectively degrade and remove disease-causing proteins.

Surrozen Expands Board of Directors with Appointment of Mary Haak-Frendscho, Ph.D.

Retrieved on: 
Thursday, March 11, 2021

SOUTH SAN FRANCISCO, Calif., March 11, 2021 (GLOBE NEWSWIRE) -- Surrozen Inc., a biotechnology company discovering and developing drug candidates to selectively modulate the Wnt pathway, today announced the addition of Mary Haak-Frendscho, Ph.D., to the Board of Directors.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., March 11, 2021 (GLOBE NEWSWIRE) -- Surrozen Inc., a biotechnology company discovering and developing drug candidates to selectively modulate the Wnt pathway, today announced the addition of Mary Haak-Frendscho, Ph.D., to the Board of Directors.
  • Dr. Haak-Frendscho brings over two decades of expertise in the discovery, translation and early development of novel therapeutics.
  • Im excited to be joining the Surrozen Board as it begins to advance novel drug candidates into clinical development and explore the full potential of Wnt biology, said Dr. Haak-Frendscho.
  • Surrozen is a biotechnology company discovering and developing drug candidates to selectively modulate the Wnt pathway.