CMTA backs ASO drug program for CMT2A with $110,500 investment

The Charcot-Marie-Tooth Association is funding a new partnership with La Jolla Labs to develop an antisense oligonucleotide therapy for CMT2A, the most common axonal form of Charcot-Marie-Tooth disease. The project aims to target the disease’s genetic root cause and could open a path for future ASO treatments across other CMT subtypes.

Why it matters: - CMT2A causes progressive muscle weakness and loss of sensation from an early age, with no approved treatment. - The project targets the underlying genetics of a specific CMT subtype, which is the kind of precision approach CMTA wants to advance across the disease. - If successful, the work could help establish a template for ASO-based therapies in other CMT subtypes.

What happened: - The Charcot-Marie-Tooth Association announced a $110,500 investment in a partnership with La Jolla Labs to develop an antisense oligonucleotide, or ASO, medicine for CMT2A. - The effort is designed as a public-private partnership. - John Svaren, PhD, chair of the CMTA Strategy to Accelerate Research Advisory Board at the University of Wisconsin-Madison, is paired with La Jolla Labs’ ASO development expertise. - The announcement came Sept. 22, 2026.

The details: - CMT2A is the most common axonal form of Charcot-Marie-Tooth disease. - Published research shows neurons affected by CMT2A have little to no natural expression of MFN1, a related gene. - The project aims to increase MFN1 expression in those neurons to offset the effects of MFN2 mutations. - CMTA identified targeted genetic therapies as a key research priority because they address the specific biology of each CMT subtype. - La Jolla Labs has worked with disease foundations, biotech companies and academic institutions on RNA therapeutics for rare diseases. - Past work includes personalized ASOs for nano-rare patients, ASO exploration in Wolfram syndrome and RNA-targeted therapeutic discovery for ALS. - Tamar Grossman, CEO of La Jolla Labs, said the company is excited to apply its mission and expertise to CMT and collaborate with the CMT research community. - Katherine Forsey, PhD, CMTA chief research officer, said published science suggests the strategy can work for CMT2A and that La Jolla Labs has the needed expertise.

Between the lines: - The investment is small compared with late-stage drug development, but it is aimed at de-risking an early scientific hypothesis before larger funding is needed. - CMTA is using its research network to connect disease biology, outside technical expertise and foundation funding in one program. - The focus on MFN1 reflects a strategy of compensating for the effect of MFN2 mutations rather than trying to broadly treat all CMT cases the same way.

What's next: - La Jolla Labs and CMTA will work to advance the ASO program toward a potential therapeutic candidate for CMT2A. - CMTA said success in this partnership could support expansion of ASO approaches into other CMT subtypes. - La Jolla Labs continues to position itself as a developer of precision RNA therapeutics for rare and complex diseases.

The bottom line: - CMTA is betting that a targeted RNA therapy can move CMT2A treatment from broad symptom management toward a gene-specific approach.