CRISPR Therapeutics and Capsida Biotherapeutics have partnered to develop gene-editing therapies for familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia. Capsida specializes in designing virus-based means of delivering therapies to specific cells, while CRISPR’s expertise lies in gene-editing technology, particularly the CRISPR gene-editing system. “Bringing together…
Partnership Targets Gene-editing Therapies for Familial ALS
Columns
Bearing Down With Equine Intent
“Welcome to my nightmare, whoa, “Welcome to my breakdown, “I hope I didn’t scare you …” If there were a “Welcome Wagon” function in the ALS community — whereby incumbent residents indoctrinated new arrivals — surely the introduction would resemble those lyrics from Alice Cooper. Grim statistics and personal…
Clene and its subsidiary Clene Nanomedicine are negotiating to increase their manufacturing capacity as they prepare to release findings — expected early next year — from a pivotal and enrolling Phase 3 trial of CNM-Au8 as an oral therapy aiming to slow amyotrophic lateral sclerosis (ALS) progression. Negotiations include the…
Columns
When Second Opinions Get Social
Lately, I’ve noticed a strange trend happening in several online ALS communities I belong to. I’ve seen an increase in posts from people who haven’t yet been diagnosed with ALS asking members of the group to essentially offer medical opinions. A typical post begins with a list of physical symptoms,…
Amylyx Pharmaceuticals is seeking approval, in Canada, of its lead candidate AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). This regulatory submission “represents a significant milestone in our efforts to develop a new treatment option for people living with ALS who have no time to wait,” Joshua Cohen, co-founder, co-CEO,…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
Levels of the protein tau found in the cerebrospinal fluid (CSF) — the transparent liquid surrounding the brain and spinal cord — can help in diagnosing amyotrophic lateral sclerosis (ALS), a study reported. Tau levels in ALS patients also correlated with a faster progression rate and poorer survival, suggesting this…
An investigational RNA molecule called an RNA aptamer halted the death of motor neurons and helped preserve motor function in a mouse model of amyotrophic lateral sclerosis (ALS). Further research is ongoing to confirm the findings in additional ALS models before testing it in humans, “but I’m optimistic that this…
On occasion, I’ll page through the photo books my husband, Todd, made for me as Christmas gifts. I feel a sense of gratitude as I look back on all we’ve been able to do since his ALS diagnosis. Family game nights. Outings to sporting events. Family movie nights. Several…
Stem cells derived from fat tissue significantly improved motor function and delayed disease onset by protecting motor neurons in a mouse model of amyotrophic lateral sclerosis (ALS), a study shows. The study, “Adipose derived stem cells protect motor neurons and reduce glial activation in both in…
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