University of Alberta's Dr. Toshifumi Yokota and Lab Achieve Top Rankings in Muscular Dystrophy Research

Edmonton, Alberta – 21 May 2024 

The University of Alberta's Dr. Toshifumi Yokota and the research team have been recognized among the top scholars in the field of muscular dystrophy research by ScholarGPS. This prestigious recognition highlights the lab's significant contributions and the impactful work being conducted at the University of Alberta.

Dr. Yokota, a professor in the Department of Medical Genetics and the holder of The Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair, has been instrumental in advancing research in muscular dystrophy. The lab's innovative approaches and dedication to improving treatments for this debilitating condition have earned a prominent position in the latest ScholarGPS rankings.

Excellence in Muscular Dystrophy Research

According to the ScholarGPS rankings, Dr. Yokota's lab is placed second in the world (Top 0.01%) for contributions to muscular dystrophy research over the past five years. This recognition is based on the number of publications, citations, and the overall impact of the work in the scientific community. The research focuses on developing therapeutic strategies, including antisense oligonucleotide therapy and genome editing, to treat muscular dystrophy and other neuromuscular diseases.

"We are honoured to be recognized among the top scholars in muscular dystrophy research," said Dr. Yokota. "This achievement is a testament to the hard work and dedication of the incredible team. We are committed to advancing our understanding of muscular dystrophy and developing effective treatments to improve the lives of patients affected by this condition."

Advancing Personalized Medicine

In addition to recognition in muscular dystrophy research, Dr. Yokota's lab has also been ranked fourth (Top 0.02%) in the field of personalized medicine. The work in this area focuses on tailoring medical treatments to individual patients based on their genetic makeup, providing more effective and targeted therapies.

"Our goal is to transform genetic discoveries into therapies that can make a real difference in patients' lives," said Dr. Yokota. "By advancing personalized medicine, we aim to develop treatments that are not only effective but also customized to the unique needs of each patient."

University of Alberta's Global Ranking

The University of Alberta itself has also earned a distinguished place in the global academic community, consistently ranked among the top universities worldwide. This recognition reflects the university's commitment to excellence in research, education, and innovation. The institution's supportive environment and state-of-the-art facilities enable researchers like Dr. Yokota and the team to make groundbreaking advancements in their fields.

Future Directions

Looking ahead, Dr. Yokota and the team are excited about the potential of ongoing research projects. The exploration of new frontiers in genetic therapy and dedication to driving innovation in the field continue. The commitment to excellence and collaboration with other leading researchers worldwide positions the lab at the forefront of scientific advancements in muscular dystrophy and personalized medicine.

The recognition from ScholarGPS underscores the University of Alberta's reputation as a leading institution for medical research. It also highlights the impactful work being done by Dr. Yokota's lab, which continues to push the boundaries of scientific knowledge and improve patient outcomes.

8 May 2024

Yokota Lab Celebrates OligomicsTx's Achievement in Securing Edmonton Edge Fund Support

In a significant boost for local biotechnology innovation, OligomicsTx, a spin-off from the Yokota Lab at the University of Alberta, has been awarded $100,000 by the Edmonton Edge Fund. This funding will propel the preclinical development of a novel treatment for Facioscapulohumeral Muscular Dystrophy (FSHD), marking a crucial step forward in combating this rare neuromuscular disorder.

FSHD, which affects approximately 1 in 8,000 people globally, leads to progressive skeletal muscle loss that initially appears in the face, shoulders, and upper arms. Despite its prevalence, no effective treatments are currently available on the market, making this research potentially transformative for patients worldwide.

The funding will facilitate critical preclinical studies, setting the stage for future clinical trials. These studies are vital for ensuring the safety and efficacy of the new genetic treatments developed by OligomicsTx, which specialize in targeting RNA to correct genetic abnormalities underlying muscular dystrophies.

The Edmonton Edge Fund aims to reduce financial barriers for innovative enterprises within the city, focusing on those poised to make significant economic or social impacts. This initiative is part of a broader strategy to foster a thriving business ecosystem in Edmonton, capable of driving technological and economic growth across various sectors.

15 March 2024

OligomicsTx Clinches $155,000 at Startup TNT Life Sciences Summit Finale

Edmonton, Alberta, March 15, 2024 — OligomicsTx, a pioneering biotech startup co-founded by University of Alberta Professor Toshifumi (Toshi) Yokota, has been awarded a significant investment of $155,000 by angel investors at the highly competitive Startup TNT Life Sciences Summit Finale. The event, which spotlighted innovation within the life sciences sector, was held on March 14 in Edmonton at the Alberta Machine Intelligence Institute.

OligomicsTx, dedicated to developing groundbreaking RNA-targeting therapies for rare neuromuscular disorders, emerged victorious against a strong lineup of contenders, including PulmVita, MACH32, Qualisure Diagnostics, Wave View Imaging, and Difinity Solutions. The company's focus on leveraging advanced genetic research to create impactful treatments underscored the event's emphasis on supporting ventures with the potential to revolutionize healthcare.

"I am deeply honored to receive this recognition and investment," said Dr. Yokota, reflecting on the win. "This achievement is a testament to the hard work and dedication of our team at OligomicsTx and the University of Alberta. We are committed to advancing our research and bringing new therapies to patients who need them most."

The summit not only highlighted OligomicsTx’s innovative approach to treating neuromuscular disorders but also facilitated valuable connections with investors interested in propelling the life sciences ecosystem forward. Side deals with PulmVita and Qualisure, though undisclosed, were also celebrated, showcasing the event's role in fostering a vibrant investment landscape for life sciences startups.

The OligomicsTx team extends its gratitude to Startup TNT, the angel investors, and all participants for their support and confidence in the company’s mission. With this investment, OligomicsTx is poised to accelerate its research and development efforts, inching closer to realizing its vision of transforming the lives of individuals affected by rare genetic conditions.

As OligomicsTx continues on its path of innovation and discovery, it remains dedicated to collaborating with researchers, healthcare professionals, and the broader community to tackle the challenges facing rare disease treatment today.

About OligomicsTx

OligomicsTx is a biotechnology company co-founded by Dr. Toshifumi Yokota, Professor of Medical Genetics at the University of Alberta. The company is at the forefront of developing RNA-targeting therapies aimed at treating rare neuromuscular disorders. Leveraging cutting-edge research and technology, OligomicsTx aspires to bring innovative and effective treatments to patients worldwide.

About Startup TNT

Startup TNT (Thursday Night Tradition) is an initiative aimed at fostering connections among startups, investors, and the broader community to support and grow the local innovation ecosystem. Through its investment summits and other activities, Startup TNT aims to facilitate funding and mentorship opportunities for emerging companies in the technology and life sciences sectors.

12 July 2023

Enhancing Canadian Neuromuscular Research and Care: NMD4C Secures 5-Year Grant from CIHR-IMHA with Additional Support from MDC

In a significant advancement for neuromuscular disease research in Canada, Dr. Toshifumi Yokota's lab, along with collaborators, has been awarded a prestigious Canadian Institutes of Health Research (CIHR) Network Grant focused on Muscular Dystrophy. This grant, part of a broader initiative that also includes Skin Health and Bone Health, allocates $200,000 CAD to the Neuromuscular Disease Network for Canada (NMD4C) for the period from July 1, 2023, to June 30, 2028.

NMD4C aims to consolidate the efforts of Canada's leading experts in neuromuscular diseases to address and surmount the current and emerging challenges in the field. As a Co-applicant, Dr. Yokota's involvement in this project underscores the pivotal role of the lab in advancing research and therapy development for muscular dystrophy and related neuromuscular disorders.

Driving Forward Muscular Dystrophy Research

The funding will enable NMD4C to further its mission to improve care, enhance research collaborations, and expedite the development of therapies for neuromuscular diseases across Canada. By fostering a collaborative network that includes clinicians, scientists, patients, and patient advocacy groups, NMD4C ensures a comprehensive approach to tackling the complexities of muscular dystrophy.

Dr. Yokota's lab, renowned for its groundbreaking work in genetic therapies, will contribute its expertise to the network's objectives. This includes developing innovative treatment strategies, improving diagnostic tools, and enhancing the understanding of the genetic and molecular mechanisms underlying neuromuscular diseases.

A Collaborative Effort Towards a Common Goal

The CIHR Network Grant recognizes the importance of interdisciplinary collaboration in driving scientific breakthroughs and improving patient outcomes. Dr. Yokota and colleagues will work closely with network partners to share knowledge, resources, and technologies. This collaborative effort aims to accelerate the pace of discovery and the translation of research findings into clinical practice.

Implications for Patients and the Future of NMD Research

The grant not only represents a significant investment in neuromuscular disease research but also signals hope for patients and families affected by muscular dystrophy. Through the concerted efforts of NMD4C, patients across Canada can look forward to advancements in care and access to cutting-edge therapies.

Dr. Yokota expressed enthusiasm for the project: "We are honored to receive this CIHR Network Grant and excited about the potential impact of our collective efforts. By working together, we can make significant strides in understanding and treating muscular dystrophy, ultimately improving the lives of those affected by these challenging conditions."

For more information about Dr. Yokota's lab and its contributions to neuromuscular disease research, please visit Dr. Yokota's lab website.

• 12 July 2023

Personalized Medicine for Rare Genetic Diseases

An interview with Dr. Toshi Yokota, a medical genetics professor at the University of Alberta, highlights the potential of personalized medicine in the treatment of rare genetic diseases. In this interview, Dr. Yokota discusses the development of custom gene therapies, inspired by the success of drugs like milasen and atipeksen, which were designed specifically for individuals with rare genetic disorders. This interview sheds light on the innovative approaches and challenges in the field of personalized medicine, offering hope for millions of people worldwide living with rare diseases. Read the full interview to explore the transformative potential of tailored therapies and the future of personalized medicine.

31 January 2023

Subcutaneous Treatment with DG9-conjugated Morpholino Shows Promising Results in SMA Mice

In a breakthrough study titled "DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration," published on January 31, 2023, researchers demonstrated a promising strategy to treat spinal muscular atrophy (SMA) and potentially other neurological diseases.

The study was conducted by a group of distinguished researchers, including Tejal Aslesh, Jun Ren, Stanley Woo, Simon Gosgnach, John Greer, and Toshifumi Yokota from the University of Alberta.

Antisense oligonucleotide (AO) therapy has shown potential in treating several neurological conditions, including SMA. However, the main challenge lies in limited delivery to the Central Nervous System (CNS) when AOs are administered intravenously or subcutaneously.

In this study, the team demonstrated that a single subcutaneous administration of a cell-penetrating peptide, DG9, conjugated to an AO known as phosphorodiamidate morpholino oligomer (PMO), reached the CNS in SMA mice models. This delivery significantly prolonged the median survival compared with unconjugated PMO and another compound R6G-PMO.

Treatment with DG9-PMO led to a substantial increase in the expression of full-length survival of motor neuron 2 in both the CNS and systemic tissues compared with non-treated and unmodified AO-treated mice. It also ameliorated the atrophic musculature and improved breathing function, muscle strength, and innervation at the neuromuscular junction, without any apparent toxicity signs.

The researchers also discovered that the DG9-conjugated PMO localized in nuclei in the spinal cord and brain after subcutaneous injections, further reinforcing the treatment's potential efficacy.

Consequently, the study identified DG9 peptide conjugation as a highly effective method to enhance the efficacy of AO-mediated splice modulation. The authors conclude that DG9-PMO represents a promising therapeutic option for SMA and other neurological diseases, overcoming the necessity for intrathecal injections and offering a means of treating body-wide tissues without apparent toxicity.

BioAlberta Recognizes Alberta Innovators and Entrepreneurs at Annual Health and Life Sciences Showcase & Awards Ceremony

September 28, 2022 11:00 AM Eastern Daylight Time

EDMONTON, Alberta--(BUSINESS WIRE)--BioAlberta announced the recipients of its 2022 Achievement Awards in recognition of the outstanding contributions of the individuals and companies whose innovation and achievements have contributed to the growing success of Alberta's life sciences sector. The awards were presented at BioAlberta’s Annual Health and Life Sciences Showcase & Awards Ceremony, held in Calgary on September 27th.

2022 Scientific Achievement and Innovation winner one – Dr. Toshifumi Yokota

“The work in Dr. Toshi’s lab has inspired a multitude of students in the area of medical genetics, collectively producing over 90 publications on their studies…He has since built on his original discovery to create a more powerful treatment with the potential to help over 40% of patients with DMD, which is preventing heart failure in patients with muscular dystrophy,” said Nuzhat Tam Zaman, Co-Founder of Sinoveda/ Co-Chair of the BioAlberta Board of Directors.

Dr. Toshifumi (Toshi) Yokota is a Professor of Medical Genetics at the University of Alberta.

Dr. Yokota's research team designed and tested synthetic DNA-like molecules called gapmers that interfere with the production of a toxic protein that destroys the muscles in facioscapulohumeral muscular dystrophy (FSHD), the third most common form of muscular dystrophy. They demonstrated that this first of a kind treatment knocked down more than 99 percent of the toxic gene products called DUX4 in patient-derived cells accompanied by morphological and functional improvement.

Accepting this award via video, Dr. Toshifumi Yokota said: “Thank you so much for this award, it means so much to me and my team. We started this laboratory 10 years ago and our therapy was FDA approved a couple of years ago. My goal is to boost the potential of this therapy because it not only cures genetic diseases but can also be used in cancer therapy and infectious diseases. I would like to end by saying a big thank you to everyone including my colleagues, partner, and Dr. Rika Yokota-Maruyama.”

About BioAlberta

BioAlberta is the voice and champion for life sciences in Alberta, committed to creating a thriving and competitive industry by facilitating and accelerating economic diversification, investment attraction and job growth.

As a private, not-for-profit industry association, BioAlberta represents more than 230 members of Alberta’s growing life sciences community of researchers, producers and suppliers operating in specialized sectors such as pharmaceuticals, medical devices, natural health products, as well as environmental, agricultural and industrial biotechnology. BioAlberta’s activities are focused on advocacy, promotion and proactively facilitating growth of the industry. Please visit: https://www.bioalberta.com/

Contacts

BioAlberta: Robb Stoddard, President and CEO
Tele: 780-425-3815
Email robb@bioalberta.com

 22 February, 2022 

Development of New Therapy Holds Promise for Duchenne Muscular Dystrophy Treatment

A study published on February 22, 2022, reports the development of a novel therapy for Duchenne muscular dystrophy (DMD), a fatal disorder characterized by progressive muscle weakness. The research, led by Kenji Rowel Q. Lim, Rika Maruyama, and Toshifumi Yokota among others, proposes a more economical approach to treating DMD that could benefit nearly half of all patients with the condition.

DMD is primarily caused by out-of-frame deletions in the dystrophin gene. These deletions lead to the absence of dystrophin, a protein essential for muscle strength and stability. At present, there is no cure for DMD. However, an emerging potential therapy called exon skipping is on the horizon. This method uses antisense oligonucleotides to convert out-of-frame mutations to in-frame mutations, enabling the production of a truncated but partially functional dystrophin.

The currently approved exon skipping therapies have limited applicability and efficacy, each treating only 8 to 14% of DMD patients. The authors of this study, however, developed a method to skip DMD exons 45 to 55, a strategy that could treat 40 to 47% of all DMD patients.

Experiments with immortalized patient myotubes showed that exons 45 to 55 could be skipped by targeting just five exons. The researchers found that conjugating a cell-penetrating peptide, DG9, to phosphorodiamidate morpholino oligomers (PMOs) improved dystrophin restoration and muscle function in mice. Local administration of a minimized exons 45 to 55–skipping DG9-PMO mixture also restored dystrophin production.

This new therapy represents a significant step forward in the potential treatment of DMD and paves the way for the development of a more economical and effective exons 45 to 55–skipping DMD therapy.

 2 December, 2021 

Duchenne Muscular Dystrophy Pig Model Provides New Opportunities for Treatment Evaluation

A collaborative study by scientists from universities across the world, including the University of Alberta, Ulster University, and Texas A&M University, reports the development of a Yucatan miniature pig model of Duchenne muscular dystrophy (DMD) with an exon 52 deletion mutation. This mutation mirrors one of the most common ones seen in DMD patients. The model was created using recombinant adeno-associated virus-mediated gene targeting and somatic cell nuclear transfer.

DMD is a lethal X-linked recessive disorder caused by mutations in the DMD gene, leading to the absence of the dystrophin protein. Treatments using phosphorodiamidate morpholino oligomer (PMO)-antisense oligonucleotides (ASOs) targeting exon 51 or 53 to reestablish the DMD reading frame have been commercially available but have limitations, with applicability only to specific patients. Hence, the development of large animal models and the evaluation of exon skipping are crucial to advance ASO development and to understand dystrophinopathies better.

The created DMD pig model exhibits dystrophin deficiency in skeletal and cardiac muscles, along with a lack of recruitment of dystrophin-associated proteins to the sarcolemma. Disease onset in these pigs was early, with severe body-wide skeletal muscle degeneration, poor growth, and physical abnormality, yet there was no obvious cardiac phenotype.

The researchers also demonstrated that primary DMD pig skeletal muscle cells with the genetically engineered exon-52 deleted pig DMD gene could evaluate the efficacy of exon 51 or 53 skipping with PMO and its advanced technology, peptide-conjugated PMO.

This study highlights the potential of the DMD pig model as a valuable resource for evaluating in vivo exon skipping efficacy, providing an instrumental tool for further DMD research and therapy development.

 23 November, 2021 

Dp71 Dystrophin Isoform Overexpression Impacts Cardiac Function: Insights from Mouse Model

Researchers from the University of Alberta recently published a study in the International Journal of Molecular Sciences revealing insights into the role of the Dp71 dystrophin isoform in Duchenne muscular dystrophy (DMD). The study was conducted by using a unique mouse model that overexpresses Dp71, enabling the researchers to understand its impact on both skeletal and cardiac muscle phenotypes.

DMD, a progressive disorder causing muscle weakness and cardiac dysfunction, is characterized by the loss of dystrophin, a 427 kDa protein vital for muscle cell membrane stability. Although multiple promoters along the dystrophin gene give rise to several shorter isoforms, including the 71 kDa Dp71, there has been a lack of strong evidence supporting Dp71's role in DMD pathology.

The study utilized del52;WT mice, which apart from the mouse Dmd gene, are heterozygous for a full-length, exon 52-deleted human DMD transgene expected to only allow Dp71 expression in muscle. This unique characteristic causes the del52;WT mice to overexpress Dp71 through both the human and murine dystrophin genes.

The team found elevated Dp71 protein levels in del52;WT mice, significantly higher in the heart than in the tibialis anterior muscle. Despite having generally normal skeletal muscle, del52;WT mice showed significant systolic dysfunction in the heart as early as three months, although no histological abnormalities were found in either the tibialis anterior or heart.

These findings suggest that overexpression of Dp71 might have more detrimental effects on cardiac function than on skeletal muscles, further highlighting the critical role of Dp71 in DMD pathogenesis. This study helps broaden our understanding of DMD and potentially opens up new avenues for developing therapeutic strategies.

SCIENCE AND TECHNOLOGY HEALTH AND WELLNESS RESEARCH

Online resource will speed up development of new approach to treating genetic diseases

U of A research team leads international effort to advance exon-skipping therapy with the help of their machine-learning-based eSkip-Finder tool and database.

Keri Sweetman - 29 June 2021

FACULTY OF MEDICINE & DENTISTRY

UNIVERSITY OF ALBERTA

• 9 June, 2021

Machine Learning-based Web Application eSkip-Finder Aids in Identifying Optimal Antisense Oligonucleotides Sequences for Exon Skipping

A study published in Nucleic Acids Research introduces eSkip-Finder, a novel web-based resource aimed at assisting researchers in identifying effective antisense oligonucleotides (ASOs) for exon skipping. This tool, developed using machine learning algorithms, could play a significant role in mRNA splicing modulation.

Exon skipping using ASOs has emerged as a powerful tool in treating genetic diseases, with several exon-skipping ASOs gaining approval worldwide. However, selecting an optimal sequence for exon skipping remains a challenging task, mainly due to the unpredictability of ASO efficacy and the multiple factors affecting exon skipping.

To address this challenge, the researchers developed eSkip-Finder, a computational tool incorporating various parameters and experimental data to design highly effective ASOs for exon skipping. eSkip-Finder provides a predictor for the exon skipping efficacy of new ASOs and a database of existing exon skipping ASOs.

The predictor enables swift analysis of exon/intron sequences and ASO lengths to identify effective ASOs for exon skipping based on a machine learning model trained with experimental data. The researchers verified that the predictions correlated well with the in vitro skipping efficacy of sequences that were not included in the training data.

Meanwhile, the database allows users to search for ASOs using queries like gene name, species, and exon number.

The eSkip-Finder tool leverages the literature data on exon skipping antisense oligonucleotides, providing both a database and a predictive tool for skipping efficacy. This helps researchers in designing effective exon skipping therapies, potentially accelerating the development of treatments for various genetic diseases.

Science Daily

Researchers developing new 'DNA stitch' to treat muscular dystrophy

Experimental treatment has potential to help almost half of patients with Duchenne muscular dystrophy

:September 25, 2019