FSHD | Jotup

Epic Bio Announces Robust Slate of Presentations at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting

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Tuesday, April 9, 2024

Poster, SAN, Cell, Abstract, Baltimore Convention Center, Gene, FSHD, Ballroom, Society, Convention, Dentistry

SOUTH SAN FRANCISCO, Calif., April 09, 2024 (GLOBE NEWSWIRE) -- Epic Bio , a leading epigenetic editing company that plans to have its FSHD program enter the clinic this year, today announced the acceptance of six abstracts, three oral presentations, and three poster presentations at the upcoming Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place May 7-11, 2024, in Baltimore, Maryland.

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SOUTH SAN FRANCISCO, Calif., April 09, 2024 (GLOBE NEWSWIRE) -- Epic Bio , a leading epigenetic editing company that plans to have its FSHD program enter the clinic this year, today announced the acceptance of six abstracts, three oral presentations, and three poster presentations at the upcoming Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place May 7-11, 2024, in Baltimore, Maryland.
Location: Baltimore at the Baltimore Convention Center, Ballroom 1, Baltimore, Maryland
Location: Baltimore at the Baltimore Convention Center, Ballroom III, Baltimore, Maryland
Location: Baltimore at the Baltimore Convention Center, Ballroom III, Baltimore, Maryland
Location: Baltimore at the Baltimore Convention Center, Exhibit Hall, Baltimore, Maryland
Location: Baltimore at the Baltimore Convention Center, Exhibit Hall, Baltimore, Maryland

Celularity Submits Request to U.S. FDA for Orphan Drug Designation for PDA-002 Asset Treating Facioscapulohumeral Muscular Dystrophy

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Wednesday, March 20, 2024

CELU, Diagnosis, Shoulder, Biologics license application, Disease, Risk, Patient, Microsoft Exchange Server, Stem cell, IND, Facioscapulohumeral muscular dystrophy, Regenerative medicine, FDA, BLA, FSHD, Muscular dystrophy, Food, Pharmaceutical industry

FLORHAM PARK, N.J., March 20, 2024 (GLOBE NEWSWIRE) -- Celularity Inc. (NASDAQ: CELU) (“Celularity”), a regenerative medicine company developing placental-derived allogeneic cell therapies and advanced biomaterial products, announced today that it has submitted a request to the U.S. Food and Drug Administration (FDA) for orphan drug designation for its off-the-shelf, placental-derived cell therapy, PDA-002, for treating Facioscapulohumeral Muscular Dystrophy (FSHD).

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FLORHAM PARK, N.J., March 20, 2024 (GLOBE NEWSWIRE) -- Celularity Inc. (NASDAQ: CELU) (“Celularity”), a regenerative medicine company developing placental-derived allogeneic cell therapies and advanced biomaterial products, announced today that it has submitted a request to the U.S. Food and Drug Administration (FDA) for orphan drug designation for its off-the-shelf, placental-derived cell therapy, PDA-002, for treating Facioscapulohumeral Muscular Dystrophy (FSHD).
Orphan drug designation is a status given to drugs that show the potential to treat, prevent or diagnose serious or life-threatening diseases that affect fewer than 200,000 people in the U.S.
Earlier, Celularity received FDA Investigational New Drug Application clearance for PDA-002 in FSHD and plans to commence a phase 1/2 study in the second half of 2024.
The trial serves as an important component for submitting a Biologics License Application (BLA) to the FDA in the future as a potential treatment for FSHD and other types of muscular dystrophy.

Satellos Bioscience Announces 2023 Year End Financial Results and Operational Highlights

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Wednesday, March 27, 2024

Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a regenerative medicine company aimed at developing therapeutics that change the way degenerative muscle diseases are treated, announced today its financial results and operational highlights for the year ended December 31, 2023.

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Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a regenerative medicine company aimed at developing therapeutics that change the way degenerative muscle diseases are treated, announced today its financial results and operational highlights for the year ended December 31, 2023.
Subsequent to the year end, on March 4, 2024, Satellos announced positive preclinical data presented at the Muscular Dystrophy Association Clinical and Scientific Conference.
Satellos also announced that SAT-3247 would be nominated as its lead development candidate with SAT-3153 becoming the backup development candidate.
Satellos’ audited financial statements for the year ended December 31, 2023, and the related management’s discussion and analysis (MD&A) will be available on SEDAR+ at www.sedarplus.ca .

Springbok Analytics Named to Fast Company's 2024 List of the World's Most Innovative Companies

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Tuesday, March 19, 2024

CHARLOTTESVILLE, Va., March 19, 2024 /PRNewswire/ - Springbok Analytics ( www.springbokanalytics.com ), a life sciences muscle analytics company, has been named to Fast Company's prestigious list of the World's Most Innovative Companies in 2024.

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CHARLOTTESVILLE, Va., March 19, 2024 /PRNewswire/ - Springbok Analytics ( www.springbokanalytics.com ), a life sciences muscle analytics company, has been named to Fast Company's prestigious list of the World's Most Innovative Companies in 2024.
Springbok has created the only technology that comprehensively measures muscle from imaging, producing personalized 3D visualizations and analyses of muscle health.
"I have made it my life's work to improve our understanding of muscles and muscle health," said Silvia Blemker, CSO and Co-Founder of Springbok Analytics.
The World's Most Innovative Companies stands as Fast Company's hallmark franchise and one of its most anticipated editorial efforts of the year.

Solid Biosciences Announces Licensing Agreement with Armatus Bio for the Use of AAV-SLB101, a Proprietary, Muscle-Targeted Capsid, in the Development of an RNAi Therapy to treat FSHD

Retrieved on:

Thursday, March 7, 2024

Liver, Duchenne, Toxicity, Inflammation, DMD, Facioscapulohumeral muscular dystrophy, DUX4, Solid, FSHD, Capsid, MicroRNA, Oxidative stress, B cell, IND, RNA interference, AAV, Atrophy, Vaccine

The AAV-SLB101 capsid has been shown in preclinical studies to have enhanced biodistribution and improved expression in muscle cells.

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The AAV-SLB101 capsid has been shown in preclinical studies to have enhanced biodistribution and improved expression in muscle cells.
Under the terms of the agreement, Solid granted Armatus a non-exclusive worldwide license to utilize AAV-SLB101 for treatment of FSHD and will provide Armatus AAV-SLB101 plasmid material, preclinical data characterizing AAV-SLB101, and manufacturing and regulatory know-how to enable development.
Armatus will be responsible for the development and commercialization of licensed products incorporating AAV-SLB101.
With this license in place, we are eager to advance our optimized ARM-201 construct as the lead candidate toward clinical evaluation.”

Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for Fourth Quarter and Full Year 2023

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Tuesday, February 27, 2024

Conference call, Cohort, Research, GSK, Fulcrum, Acceleron, REACH, SCD, Conference, Therapy, Collaboration, FSHD, Webcast, Patient, Disease, Losmapimod, Pharmaceutical industry

CAMBRIDGE, Mass., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (“Fulcrum”) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today reported financial results for the fourth quarter and full year of 2023 as well as an update to the business.

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Collaboration Revenue: Collaboration revenue was $0.9 million for the fourth quarter of 2023 as compared to $0.7 million for the fourth quarter of 2022.
G&A Expenses: General and administrative expenses were $9.9 million for the fourth quarter of 2023 as compared to $10.1 million for the fourth quarter of 2022.
Net Loss: Net loss was $24.8 million for the fourth quarter of 2023 as compared to $26.1 million for the fourth quarter of 2022.
ET to review the fourth quarter and full year 2023 recent business highlights and financial results.

Satellos Presents Positive Preclinical Efficacy Data for SAT-3247 at the 2024 MDA Clinical & Scientific Conference

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Monday, March 4, 2024

Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preclinical data showing SAT-3247 can improve skeletal muscle function in multiple mouse models of muscle degeneration.

Key Points:

Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preclinical data showing SAT-3247 can improve skeletal muscle function in multiple mouse models of muscle degeneration.
In all instances, treatment with SAT-3247 over a three-to-four-week period resulted in a statistically significant improvement in muscle force versus animals receiving placebo.
We believe the muscle injury data further expand and broaden the potential for SAT-3247 into non-dystrophy disease indications as well as multiple forms of muscle injury or trauma in otherwise healthy individuals.
We continue on our development plan to advance SAT-3247 into first-in-human clinical trials mid-year.”
These data are being presented in a poster at the 2024 MDA Clinical & Scientific Conference being held March 3-6 in Orlando.

Avidity Biosciences Honors Rare Disease Day®

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Thursday, February 29, 2024

Rare disease, Therapy, SAN, Avidity, Caregiver, DMD, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy, Hope, Learning, DM1, RNA, Rare Disease Day, FSHD, Patient, Disease, AOC, Myopathy, Research

"Today, on Rare Disease Day, we are proud to join the global community in raising awareness for people living with rare diseases," said Sarah Boyce, president and chief executive officer at Avidity.

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"Today, on Rare Disease Day, we are proud to join the global community in raising awareness for people living with rare diseases," said Sarah Boyce, president and chief executive officer at Avidity.
"Recently, we had the privilege of hosting individuals affected by rare muscle diseases at Avidity.
Rare Disease Day takes place on the last day of February each year with the goal to raise awareness of the impact of rare diseases worldwide.
EURORDIS established Rare Disease Day in 2008 and coordinates with more than 70 national alliance patient organizations each year to honor those living with rare diseases as well as their families and caregivers.

Avidity Biosciences Announces 2024 Corporate Priorities and Catalysts for Next Stage of Growth

Retrieved on:

Friday, January 5, 2024

RNA, Marketing, Cardiology, Facioscapulohumeral muscular dystrophy, Research, DM1, Duchenne muscular dystrophy, SAN, Facial muscles, Therapy, FSHD, AOC, University, DSM-IV codes, Myotonia, Growth, Safety, Senior, Nursing

SAN DIEGO, Jan. 5, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced its 2024 corporate priorities and catalysts for the next stage of growth. In mid-2024, Avidity plans to initiate the global Phase 3 HARBOR™ trial of AOC 1001 for adults living with myotonic dystrophy type 1 (DM1). The robust data package of AOC 1001 from the Phase 1/2 MARINA® trial and open-label extension study, MARINA-OLE™, has demonstrated consistent improvements across multiple functional endpoints including myotonia, muscle strength and mobility, and long-term favorable safety and tolerability results in people living with DM1. Avidity also plans to report data in 2024 from all three of its ongoing clinical programs targeting three distinct rare muscle diseases: DM1, Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44) and facioscapulohumeral muscular dystrophy (FSHD) while advancing its cardiology programs toward clinical development. In addition, Avidity announced the appointment of Eric B. Mosbrooker as Chief Strategy Officer.

Key Points:

In mid-2024, Avidity plans to initiate the global Phase 3 HARBOR™ trial of AOC 1001 for adults living with myotonic dystrophy type 1 (DM1).
In addition, Avidity announced the appointment of Eric B. Mosbrooker as Chief Strategy Officer.
Prior to joining Avidity, Mr. Mosbrooker served as the Chief Operations Officer for Cognoa, where he spearheaded commercial initiatives, program management, product development, and business operations.
Mr. Mosbrooker holds a Bachelor of Science in Industrial Engineering from the University of Wisconsin – Madison.

Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients Demonstrating Promise of the FORCE™ Platform in Developing Therapeutics for Rare Muscle Diseases

Retrieved on:

Wednesday, January 3, 2024

“We are excited that Dyne’s first clinical data in two programs have demonstrated proof-of-concept and validated the promise of the FORCE™ platform in developing targeted therapeutics for rare muscle diseases.

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“We are excited that Dyne’s first clinical data in two programs have demonstrated proof-of-concept and validated the promise of the FORCE™ platform in developing targeted therapeutics for rare muscle diseases.
Dyne anticipates providing its next clinical data update from the ACHIEVE trial in the second half of 2024.
Key initial findings from DELIVER include:
Muscle delivery: DYNE-251 showed a mean 657 ng/g PMO muscle drug concentration at 6 months.
Dyne anticipates providing its next clinical data update from the DELIVER trial in the second half of 2024.