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WALTHAM, Mass., June 24, 2024 (GLOBE NEWSWIRE) — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle illness firm targeted on advancing modern life-transforming therapeutics for folks dwelling with genetically pushed illnesses, at present introduced new preclinical information in a Pompe illness mannequin demonstrating the potential of the FORCE™ platform to ship enzyme substitute remedy to cardiac and skeletal muscle and the central nervous system (CNS). The info have been offered on the New Instructions in Biology and Illness of Skeletal Muscle Convention, being held June 23-26 in Fort Lauderdale, FL.
“These preclinical information present for the primary time the power of FORCE to ship enzymes to skeletal and cardiac muscle in addition to the CNS, increasing the modularity of our platform past oligonucleotides. The info assist the potential of FORCE to allow efficient enzyme substitute remedy with the chance to considerably enhance upon obtainable therapies for Pompe illness,” Oxana Beskrovnaya, Ph.D., chief scientific officer of Dyne. “We sit up for persevering with to discover this software of our platform as a part of our mission to ship life-transforming therapies for folks with critical muscle illnesses.”
Pompe illness is a uncommon, extreme neuromuscular dysfunction attributable to deficiency of the lysosomal enzyme, acid alpha glucosidase (GAA). Lack of GAA results in glycogen accumulation and improve in lysosomal dimension in muscle and subsequent weak point, cardiomyopathy and respiratory failure. Enzyme substitute remedy with GAA is the usual of care and will increase survival however has insufficient efficacy in skeletal muscle. Pompe can be characterised by CNS manifestations, together with behavioral and cognitive deficits attributable to glycogen accumulation in CNS cells, which aren’t addressed by the usual of care remedy.
Dyne engineered FORCE-GAA by leveraging the FORCE platform and evaluated efficacy in vivo utilizing hTfR1/6Neo mice, that have been developed by crossing the well-established 6Neo mouse mannequin of Pompe with mice expressing human transferrin receptor 1. Intravenous administration of FORCE-GAA cleared glycogen in muscle and the CNS and normalized lysosomal dimension in hTfR1/6Neo mice. FORCE-GAA lowered serum neurofilament mild chain, a biomarker of axonal damage, offering proof of profit within the CNS. FORCE-GAA additionally displayed superior efficacy and dose efficiency in comparison with GAA alone. Extra information with FORCE-GAA confirmed the potential for month-to-month dosing which is much less frequent than permitted enzyme substitute therapies.
The presentation entitled, “FORCE™ Platform for the Improvement of Focused Therapeutics for Uncommon Muscle Illnesses” is obtainable within the Scientific Publications & Displays part of Dyne’s web site at https://www.dyne-tx.com/our-forcetm-publications/.
About Pompe Illness
Pompe illness is a extreme neuromuscular dysfunction attributable to a deficiency of the lysosomal enzyme, acid alpha glucosidase (GAA). Lack of GAA causes glycogen accumulation in tissue resulting in muscle weak point, cardiomyopathy, respiratory failure, and central nervous system (CNS) manifestations. Pompe illness belongs to a bunch of illnesses often known as the lysosomal storage problems (LSDs). It will probably current as infantile-onset, essentially the most extreme type of the illness with early onset of signs in infancy that quickly progress, or late-onset, which progressively damages muscular tissues over time. An estimated 5,000 to 10,000 people worldwide are affected by Pompe.
In regards to the FORCE™ Platform
The proprietary FORCE™ platform drives Dyne’s efforts to develop focused, trendy oligonucleotide therapeutics with the potential to be life-transforming for sufferers with critical muscle illnesses. Dyne designed the FORCE platform utilizing its deep information of muscle biology and oligonucleotide therapeutics to beat the present limitations in supply to muscle tissue with the aim of stopping or reversing illness development. The FORCE platform leverages the significance of transferrin receptor 1 (TfR1) in muscle biology as the muse for its novel method. TfR1, which is very expressed on the floor of muscle cells, is required for iron transport into muscle cells. Dyne hyperlinks therapeutic payloads to its TfR1-binding fragment antibody (Fab) to develop focused therapeutics for muscle illnesses.
About Dyne Therapeutics
Dyne Therapeutics is a clinical-stage muscle illness firm targeted on advancing modern life-transforming therapeutics for folks dwelling with genetically pushed illnesses. With its proprietary FORCE™ platform, Dyne is growing trendy oligonucleotide therapeutics which are designed to beat limitations in supply to muscle tissue. Dyne has a broad pipeline for critical muscle illnesses, together with medical applications for myotonic dystrophy kind 1 (DM1) and Duchenne muscular dystrophy (DMD) and a preclinical program for facioscapulohumeral muscular dystrophy (FSHD). For extra info, please go to https://www.dyne-tx.com, and comply with us on X, LinkedIn and Fb.
Ahead-Wanting Statements
This press launch comprises forward-looking statements that contain substantial dangers and uncertainties. All statements, apart from statements of historic info, contained on this press launch, together with statements concerning Dyne’s technique, future operations, prospects and plans, goals of administration, the potential of the FORCE platform, the anticipated timelines for reporting further information for FORCE-GAA, expectations concerning the initiation of further preclinical research or medical trials of FORCE-GAA, and plans to offer future updates on pipeline applications, represent forward-looking statements inside the which means of The Personal Securities Litigation Reform Act of 1995. The phrases “anticipate,” “imagine,” “proceed,” “might,” “estimate,” “count on,” “intend,” “could,” “would possibly,” “goal,” “ongoing,” “plan,” “predict,” “venture,” “potential,” “ought to,” or “would,” or the unfavourable of those phrases, or different comparable terminology are supposed to determine forward-looking statements, though not all forward-looking statements include these figuring out phrases. Dyne could not really obtain the plans, intentions or expectations disclosed in these forward-looking statements, and you shouldn’t place undue reliance on these forward-looking statements. Precise outcomes or occasions might differ materially from the plans, intentions and expectations disclosed in these forward-looking statements because of numerous vital elements, together with: uncertainties inherent within the identification and growth of product candidates, together with the initiation and completion of preclinical research and medical trials; uncertainties as to the provision and timing of outcomes from preclinical research and medical trials; the timing of and Dyne’s potential to enroll sufferers in medical trials; whether or not outcomes from preclinical research and preliminary information from early medical trials will likely be predictive of the ultimate outcomes of the medical trials or future trials; whether or not Dyne’s money sources will likely be adequate to fund its foreseeable and unforeseeable working bills and capital expenditure necessities; in addition to the dangers and uncertainties recognized in Dyne’s filings with the Securities and Change Fee (SEC), together with the Firm’s most up-to-date Kind 10-Q and in subsequent filings Dyne could make with the SEC. As well as, the forward-looking statements included on this press launch characterize Dyne’s views as of the date of this press launch. Dyne anticipates that subsequent occasions and developments will trigger its views to alter. Nevertheless, whereas Dyne could elect to replace these forward-looking statements in some unspecified time in the future sooner or later, it particularly disclaims any obligation to take action. These forward-looking statements shouldn’t be relied upon as representing Dyne’s views as of any date subsequent to the date of this press launch.
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