Genomics is an ever-evolving field that demands cutting-edge education initiatives and student preparation strategies. NHGRI’s Short Course in Genomics is one of these educational pathways designed to realize its vision of improving human health through advances in genomic research while making sure these benefits reach all communities.
Applications are reviewed by a panel of NHGRI employees from across subject areas across the Institute, to create an even distribution of educators.
Enrollment Dates
Wave Life Sciences is a clinical-stage genetic medicines company focused on unlocking the full potential of RNA medicines. Through its proprietary discovery and development platform PRISMTM, Wave uses multiple modalities and chemical innovation to address complex diseases with unmet medical needs. Wave’s pipeline currently features clinical programs targeting Duchenne muscular dystrophy, alpha-1 antitrypsin deficiency, Huntington’s disease as well as obesity preclinically.
MaRS Discovery District in Toronto hosts several research hospitals, medical research institutes, AI research labs from Google, Uber and Vector; Pfizer, Takeda Pharmaceuticals and GSK are major investors. To date, MaRS Health Technologies has raised more than $102 million through private investors with Pfizer being one of them.
Wave’s experimental Duchenne drug failed in clinical trials last year and two treatments for Huntington’s were discontinued, yet Wave remains committed to its mission of becoming a leader in genetic medicine.
Enrollment Options
Wave Genetics, which utilizes biotechnology to manipulate DNA’s holographic patterning, has immense potential in terms of organ regeneration, human lifespan expansion, genetic decoding and deciphering.
Dr Peter Gariaev and his scientific team have devised an ingenious system to communicate with your DNA at its quantum level and assist it in correcting itself – this revolutionary idea has been proven in clinical studies. Esoteric and spiritual teachers have long recognized how our bodies can be programmed using thoughts, language and emotions; now science has shown how our DNA can also be programmed through sound waves.
Wave’s flagship program, WVE-210201, is a GalNAc-conjugated small interfering RNA (siRNA), targeting INHBE. This siRNA aims to reduce expression of this gene to prevent obesity and enhance cardiometabolic health. In 2025, the company plans to select clinical candidates for PNPLA3, LDLR and APOB as part of their splice correction programs; additionally they intend to further their Duchenne muscular dystrophy (DMD) candidate targeting exon 51. Wave is located in Toronto’s MaRS Discovery District, home to four research hospitals and institutes as well as AI research labs belonging to Google, Uber and Vector. Their experienced management team has attracted several investors such as GSK Ventures Horizons Innovation Fund Ontario Capital Investment Corporation as well as Vector Institute for Artificial Intelligence as investors.
Registration
Wave Life Sciences is a clinical-stage genetic medicines company dedicated to unlocking the vast potential of RNA medicine to transform human health. Their unique PRISMTM platform combines multiple techniques such as editing, splicing and antisense for scientific breakthroughs that treat both rare and prevalent disorders alike. Their diverse portfolio currently includes programs targeting Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and Huntington’s disease as well as preclinical programs in obesity treatment.
Add Health offers unique longitudinal data capturing biological, psychosocial and environmental information about nationally representative samples of siblings from early adolescence into adulthood. The in-home sample is composed primarily of full siblings recruited through an in-school survey and supplemented by random selection of half-siblings and unrelated adolescents such as step-siblings, adopted children or those living in group homes. Additional sibling samples are drawn based on responses to an in-school survey which indicates that they live with one of their brothers or sisters who has already been included in the study. Furthermore, Add Health eScience Center recruits teens as a population cohort of twins for further investigation.
Geno4ME utilized an online, digital recruitment and consent process to invite participants into its program. Once participants agreed, they received a saliva kit for DNA collection with pre-paid shipping labels to return their kits back to clinical laboratories for processing, then were contacted by telephone with results.
Geno4ME found that digital outreach strategies were particularly successful at engaging patients proactively in WGS research programs. As more healthcare systems adopt population-level WGS programs, understanding which demographic groups prefer digital recruitment approaches will become even more crucial to increasing enrollment rates, especially among populations underrepresented in genomics.
Wave Pharmaceuticals started dosing healthy volunteers as part of its RestorAATion trial for AATD in December 2023. This Phase 1b/2a trial seeks to assess whether WVE-006 administration alone restores M-AAT levels in blood, providing crucial initial evidence regarding its mechanism and efficacy as an antiaattridial agent.
