YOLT-201 therapy for FAP, other forms of ATTR gets funding boost
$45 in financing will fund additional clinical programs, development plans
Yoltech Therapeutics has raised about $45 million in funding to advance its clinical programs, including YOLT-201, an experimental gene-editing therapy for familial amyloid polyneuropathy (FAP) and other forms of transthyretin amyloidosis (ATTR).
YOLT-201 is being tested in a Phase 1/2a clinical trial (NCT06539208) that is still recruiting patients with a genetic diagnosis of FAP or ATTR with cardiomyopathy (ATTR-CM), a form of the disease that mostly affects the heart, at three locations in China. Another investigator-led Phase 1 clinical trial (NCT06082050) is testing YOLT-201 in ATTR-CM regardless of whether it has a hereditary basis.
Financing from this Series B round, led by the AstraZeneca-CICC healthcare investment fund, a private equity fund born out of a partnership between AstraZeneca and China International Capital Corporation (CICC), will also support other clinical programs and plans for global development.
“This financing marks an important milestone as we continue advancing our pipeline toward transformative therapies. We are committed to turning cutting-edge gene-editing science into real-world treatments for patients worldwide,” Yuxuan Wu, Yoltech’s founder and CEO, said in a company press release.
YOLT-201 uses CRISPR gene-editing system to modify TTR gene
ATTR can be hereditary, meaning it is passed down through families due to a genetic mutation in the TTR gene, or wild-type, which is when it develops sporadically with aging. In both cases, the protein transthyretin misfolds and builds up as toxic clumps, damaging the body’s tissues.
In FAP, also known as hereditary ATTR with polyneuropathy, the toxic clumps tend to build up in the multiple peripheral nerves that extend outside the brain from the spinal cord to the body’s extremities, leading to widespread nerve damage and neurological symptoms.
YOLT-201 is given as a single infusion into the bloodstream and uses the CRISPR gene-editing system to modify the TTR gene and block production of the faulty transthyretin protein. The therapy is carried to liver cells — where TTR is normally produced — by lipid nanoparticles, tiny fat-based particles that act as delivery vehicles. This is expected to stop toxic clumps from forming, thereby easing symptoms.
Two-part clinical trial tests therapy’s safety, efficacy
Cleared earlier last year by Chinese regulators, the ongoing two-part clinical trial is testing how safe YOLT-201 is and how well it works in adults with FAP or ATTR-CM. It is also testing the pharmacokinetics of YOLT-201 — that is, how it moves into, through, and out of the body.
In the first part of this open-label clinical trial, YOLT-201’s dose is increased in different groups of patients until the highest dose with the lowest risk profile is found. The second part will test the safety and efficacy of the optimal biological dose in a larger number of patients.
Early data from six adults with FAP showed that a single infusion of YOLT-201 reduced circulating levels of disease-causing transthyretin protein by more than 90% at the higher of the two doses tested — without causing any serious side effects or dose-limiting toxicities.
In the investigator-led Phase 1 clinical trial, the therapy significantly reduced circulating transthyretin in seven adults with ATTR-CM. The most common side effects were infusion-related reactions and transient increases in liver enzymes. Two patients received a second infusion to achieve the target reduction in circulating transthyretin.
YolTech is also developing YOLT-101 for familial hypercholesterolemia, a hereditary disease marked by very high cholesterol levels. YOLT-101 is in Phase 1 clinical testing (NCT06461702 and NCT06458010) in China. The company’s pipeline also includes YOLT-204 for beta-thalassemia/sickle cell disease.
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