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Shark Tooth Biotech

@SharkToothBio

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Developing treatments for CMT1A. Follow us to learn more about the science behind CMT1A, and potential pathways to treatments.

San Francisco
Joined July 2024
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@SharkToothBio
Shark Tooth Biotech
1 month
RT @davidcapple: Big thanks to @fishmanaf for inviting me to join him on the Startup Dad podcast. This conversation was deeply personal anโ€ฆ.
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@SharkToothBio
Shark Tooth Biotech
7 months
RT @davidcapple: ๐—œ๐—ป๐˜๐—ฟ๐—ผ๐—ฑ๐˜‚๐—ฐ๐—ถ๐—ป๐—ด @SharkToothBio: ๐—” ๐—ฃ๐—ฒ๐—ฟ๐˜€๐—ผ๐—ป๐—ฎ๐—น ๐— ๐—ถ๐˜€๐˜€๐—ถ๐—ผ๐—ป ๐˜๐—ผ ๐—–๐—ผ๐—บ๐—ฏ๐—ฎ๐˜ ๐—–๐— ๐—ง๐Ÿญ๐—”. 2 years ago, my world was flipped upside down. My 2-year-oโ€ฆ.
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@SharkToothBio
Shark Tooth Biotech
7 months
This 2024 study reveals that boosting cGMP with drugs like tadalafil or CYR119 improves proteostasis, myelination, and nerve conduction in mouse models of #CMT1A & #CMT1B. Promising steps toward potential treatments!.
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@SharkToothBio
Shark Tooth Biotech
9 months
Grateful to Effie (@OnceUponAGene) for this great episode about choosing friends as rare disease parents ๐Ÿ‘‡. Highlights:.- Find your people.- Remember who you were before rare disease.- Be intentional.
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@SharkToothBio
Shark Tooth Biotech
10 months
Interesting targeting approach: Antibody-targeted chromatin delivers CRISPR/Cas9 efficiently into cells. High precision, low toxicity, and no viral components. Could this be applicable to CMT1A?.
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@SharkToothBio
Shark Tooth Biotech
11 months
Fascinating treatment for SMA ๐Ÿ‘‡. EVRYSDI is a small molecule taken daily. It modifies SMN2 gene splicing by binding to SMN2 mRNA and results in increasing the production of the life-saving SMN protein. Wondering if it could be engineered to bind to PMP22 mRNAโ€ฆ.
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@SharkToothBio
Shark Tooth Biotech
1 year
Boosting cGMP levels with sildenafil restores proteasome function, reduces proteotoxic stress, & improves myelination in a mouse model of CMT1B. This could also work for CMT1A.
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@SharkToothBio
Shark Tooth Biotech
1 year
This paper shows that we can measure a 1.55x increase in PMP22 from CMT1A skin biopsies. This implies that skin biopsies can be used to accurately measure a (future) treatment's ability to downregulate the body's production of PMP22.
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@SharkToothBio
Shark Tooth Biotech
1 year
The combination of these processes explains why CMT1A can present with early-onset symptoms that progressively worsen over time. The initial poor quality of myelin affects nerve function early in life, and continued damage to this already weak myelin exacerbates the condition.
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@SharkToothBio
Shark Tooth Biotech
1 year
Demyelinating vs. Dysmyelinating? From our reading so far it appears that CMT1A is a combination of both. Initially, myelin is improperly formed (dysmyelination). Over time, this already defective myelin becomes further damaged and degraded (demyelination).
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@SharkToothBio
Shark Tooth Biotech
1 year
Note: this study was performed on CMT1 patients (both CMT1A and 1B). Unfortunately it does not differentiate between the two groups so it's unknown how many were 1A vs. 1B.
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@SharkToothBio
Shark Tooth Biotech
1 year
Interesting chart of nerve conduction velocity over time in 107 patients with CMT.(Source:
Tweet media one
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@SharkToothBio
Shark Tooth Biotech
1 year
Excessive PMP22 disrupts the delicate balance of lipid-to-protein ratio required for proper myelin structure. This imbalance leads to the formation of disordered myelin-like assemblies, contributing to the defective myelin in CMT1A.
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@SharkToothBio
Shark Tooth Biotech
1 year
Note: there are concerns with AAV (viral vectors) delivery:.1) Since CMT1A is non-lethal, the risk-reward might not be there for some patients. 2) Taking an AAV treatment now might preclude patients from taking a safer AAV treatment in the future.
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@SharkToothBio
Shark Tooth Biotech
1 year
This study concludes that AAV2/9 gene therapy is an effective approach for treating CMT1A by reducing PMP22 overexpression in Schwann cells. This approach maintained motor and sensory functions in animal models over an extended period.
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@SharkToothBio
Shark Tooth Biotech
1 year
Note: this is not the same delivery as LNPs. Squalenoyl siRNA: chemical conjugation of siRNA to squalene, leading to self-assembly into nanoparticles. LNPs: encapsulation of siRNA within a lipid matrix, without direct chemical bonding between the siRNA and lipid components.
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@SharkToothBio
Shark Tooth Biotech
1 year
This study demonstrates that squalenoyl siRNA PMP22 nanoparticles treat CMT1A in mice, offering hope for developing a similar therapeutic approach for human patients.
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@SharkToothBio
Shark Tooth Biotech
1 year
Evidence suggests that CMT1A involves developmental defects in myelination, leading to uniformly slowed nerve conduction, rather than active demyelination. This insight challenges traditional views and impacts therapeutic approaches.
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@SharkToothBio
Shark Tooth Biotech
1 year
Is CMT1A demyelinating or dysmyelinating?.
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