QurAlis Archives - Medical Design Sourcing

QRL-201, a novel therapy targeting STMN2 expression, enters phase 1 ALS trial

Cambridge, Massachusetts-based QurAlis has dosed the first patient with the biotherapeutic QRL-201 in a Phase 1 clinical trial (ANQUR) in Canada. QRL-201 is a novel therapy targeting the restoration of STATHMIN-2 (STMN2) expression in amyotrophic lateral sclerosis (ALS) patients. QurAlis believes ANQUR the first study to evaluate such a treatment.

Stathmin-2 (STMN2), also known as superior cervical ganglia neural specific 10 (SCG10), belongs to the stathmin protein family involved in the regulation and dynamics of microtubules. Predominantly expressed in neurons, STMN2 serves key functions in neuronal development, axonal growth and regeneration.

Recent research has revealed the significance of STMN2 in neurodegenerative diseases, especially ALS and frontotemporal dementia (FTD). Researchers have linked the pathogenesis of those conditions to the loss of STMN2 function.

“In mice, STMN2 deletion causes axonal degeneration and loss of muscle innervation, w…

QurAlis targets UNC13A RNA mis-splicing in ALS and FTD with novel FlexASO platform

QurAlis, a privately-held company specializing in precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, has debuted its latest program. This initiative targets UNC13A RNA mis-splicing, a critical gene alteration present in ALS and frontotemporal dementia (FTD). FTD has recently gained renewed attention following the disclosure of Bruce Willis’ diagnosis of the condition. UNC13A is a vital regulator of neurotransmitter release at synapses, and the genetic alteration resulting in mis-splicing is estimated to occur in 58% of ALS patients and up to half of FTD cases.

To tackle this gene alteration, QurAlis has developed its proprietary FlexASO splice modulator platform. This technology uses antisense oligonucleotides (ASOs) to correct mis-splicing, restore UNC13A protein production, and reduce cryptic exons that may contribute to disease progression. Kasper Roet, QurAlis CEO, explained that “FlexASOs operate on the s…

QurAlis to tap $88M funding to develop ALS product candidates QRL-201 and QRL-101

QurAlis, a clinical-stage biotech focused on amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, announced that it has closed an oversubscribed $88 million Series B financing.

According to Kasper Roet, CEO and co-founder of QurAlis, the funding round recognizes the company’s scientific track record. “We are breaking down scientific barriers in our quest to bring much-needed precision therapies to patients,” Roet said in a statement.

In an interview at the JP Morgan Healthcare Conference earlier this year, Roet said he has long wanted to develop therapies for neurodegenerative diseases. AI-based advances have helped shine a light on the genetic underpinnings of ALS. “There’s about 40 different genes that have been now identified that contribute to development of the disease,” Roet said. The continued breakthroughs helped convince Roet that the disease was “starting to become a tractable problem,” he said. “I think we can actuall…

QurAlis homes in on drug candidates for subset of ALS patients

The Cambridge, Massachusetts–headquartered biotech QurAlis kicked the year off by launching a Phase 1 clinical study of QRL-101 (QRA-244), a potentially novel selective Kv7.2/7.3 ion channel opener to treat hyperexcitability-induced disease progression in amyotrophic lateral sclerosis (ALS).

QRL-101 targets Kv7.2/7.3 potassium channels. Kv7 modulation can potentially lower spinal and cortical motor neuron excitability, potentially improving ALS patient survival.

QurAlis company is building a pipeline of antisense oligonucleotides (ASOs) and small molecule programs for ALS subtypes that constitute the majority of ALS cases. In addition to QRL-101, its pipeline includes QRL-201, QRL-203, QRL-204 and QRL-202.

QRL-201 is also the focus of a Phase 1 trial.

The company’s QR43 Platform facilitates the discovery and development of novel therapies for TDP-43 pathologies, which are involved in some patients with ALS, frontotemporal degeneration and Alzheime…

QurAlis homes in on drug candidates for subset of ALS patients

The Cambridge, Massachusetts–headquartered biotech QurAlis kicked the year off by launching a Phase 1 clinical study of QRL-101 (QRA-244), a potentially novel selective Kv7.2/7.3 ion channel opener to treat hyperexcitability-induced disease progression in amyotrophic lateral sclerosis (ALS).

QRL-101 targets Kv7.2/7.3 potassium channels. Kv7 modulation can potentially lower spinal and cortical motor neuron excitability, potentially improving ALS patient survival.

QurAlis company is building a pipeline of antisense oligonucleotides (ASOs) and small molecule programs for ALS subtypes that constitute the majority of ALS cases. In addition to QRL-101, its pipeline includes QRL-201, QRL-203, QRL-204 and QRL-202.

QRL-201 is also the focus of a Phase 1 trial.

The company’s QR43 Platform facilitates the discovery and development of novel therapies for TDP-43 pathologies, which are involved in some patients with ALS, frontotemporal degeneration and Alzheime…

QurAlis bolsters its executive team with three new leaders

Cambridge, Massachusetts–based biotech QurAlis Corp., has appointed Bryan Boggs as head of regulatory affairs. In addition, Christopher Gerry Lohan will join as head of clinical operations, and Guzide Adhikari will take on the role of head of global supply chain management.

The company is focused on developing novel treatments for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets.

The three new hires will “play a critical role as we advance our deep pipeline of novel therapeutics for ALS and beyond to help patients desperately in need of treatment options,” said QurAlis CEO Kasper Roet in a statement.

Boggs joins QurAlis from Eli Lilly, where he worked for more than 30 years. His most recent role at the company was director, global regulatory and pharmacovigilance.

Lohan has more than 20 years of clinical research experience. In his last role, he worked as the director of clinical operation…

QurAlis selects development candidate for ALS

Privately-held biotech QurAlis plans to begin clinical development for the first-in-class molecule QRL-201 for amyotrophic lateral sclerosis (ALS) in the second half of 2022.

QRL-201 is now the subject of IND-enabling studies.

The drug candidate targets the restoration of STATHMIN-2 (STMN2) expression in ALS. STMN2 is a protein encoded by the STMN2 gene that is involved in neural repair. ALS is often associated with decreased expression of STMN2.

In ALS patient-derived motor neuron disease models with TDP43 pathology, QRL-201 restored STMN2 loss of function.

A 2008 study in Current Opinion in Neurology found that TDP43 is involved in ALS and frontotemporal dementia (FTD) pathogenesis.

TDP43 pathology is also present in some Alzheimer’s and Parkinson’s disease patients.

“QRL-201 could potentially benefit ALS patients who have a loss of STMN2 due to TDP43 pathology, which could, in turn, slow disease progression,” explained Kasper Roet…