Our Approach

Table Of Content

• Ex­clu­sive: In $1B+ bet on AI, bio­phar­ma heavy­weights back new start­up to up­end drug R&D
• Small Molecule
• Design Therapeutics Highlights Upcoming Milestones and Reports Second Quarter 2022 Financial Results
• Data to be Presented during the 2021 Virtual Myotonic Dystrophy Foundation Annual Conference
• Friedreich Ataxia Program

Xaira was co-founded by Dr. David Baker, Professor of Biochemistry and Director of the Institute for Protein Design at the University of Washington School of Medicine. The company now employs a group of researchers who developed the leading models for protein and antibody design, RFdiffusion and RFantibody, while in Dr. Baker’s lab. Xaira’s broader team will advance these models and develop new methods that can connect the world of biological targets and engineered molecules to the human experience of disease. DT-216 Phase 1 MAD Trial DesignThe Phase 1 MAD clinical trial is a randomized, double-blind, placebo-controlled study designed to evaluate multiple ascending doses of DT-216 administered intravenously in adult patients with FA. The primary and secondary study objectives were to evaluate safety and tolerability, and pharmacokinetics (PK) of three weekly doses of DT-216 in FA patients.

Ex­clu­sive: In $1B+ bet on AI, bio­phar­ma heavy­weights back new start­up to up­end drug R&D

Discovery efforts for multiple other small molecule genomic medicines are also underway. We believe our experiences with GeneTAC™ molecules allow us to more rapidly design GeneTAC™ molecules for additional indications. Our DM1 GeneTAC™ small molecules were shown to reduce observable CUG nuclear foci and correct splicing defects in DM1 patient-derived myotubes. We are working toward selection of a development candidate in anticipation of a future IND submission. Fuchs Endothelial Corneal Dystrophy (FECD) is a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells (CECs) and progressive loss of vision. Typically, the disease manifests after age 40 and can be detected through routine eye exams.

Small Molecule

Design believes these data support use of the muscle FXN mRNA assay as a sensitive indicator of clinical activity in FA, with the ability to discriminate clinically meaningful changes in endogenous FXN mRNA. Dr. Jeffries brings over twenty years of experience in biopharma with extensive experience in strategy and R&D. Prior to Design, Dr. Jeffries led projects to develop R&D and therapeutic strategies for biopharma at the Boston Consulting Group. He was instrumental in pioneering miniPCR, which performed the first PCR on the international space station.

Design Therapeutics Highlights Upcoming Milestones and Reports Second Quarter 2022 Financial Results

Design Therapeutics divulges new compounds for treatment of Huntington's disease - BioWorld Online

Design Therapeutics divulges new compounds for treatment of Huntington's disease.

Posted: Tue, 24 Oct 2023 07:00:00 GMT [source]

These foci inhibit the ability of MBNL1 to process pre-mRNAs, which when mis-spliced disrupt muscle development and function that is characteristic of DM1. Design Therapeutics, Inc a biopharmaceutical company, researches, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. As a result, the DM1 GeneTAC™ molecules are designed to prevent the formation of the CUG hairpin structures that trap splicing proteins and produce nuclear foci. As with our other programs, the DM1 program is designed to address the underlying cause of the disease and benefit from the favorable development advantages of small molecules. Pratik Shah, Ph.D., is co-founder, president, chief executive officer and chairperson of Design Therapeutics.

There are currently no approved therapies that can reverse or slow down the course of HD. Friedreich ataxia (FA) is a devastating monogenic, autosomal recessive progressive disease caused by low levels of endogenous Frataxin (FXN) due to abnormally expanded GAA triplet repeat expansions in the first intron of the FXN gene. The disease is characterized by spinocerebellar ataxia, dysarthria, pyramidal weakness, deep sensory loss, hypertrophic cardiomyopathy, skeletal abnormalities and diabetes mellitus. Clinical onset occurs most often around puberty, leading to severe disability by early adulthood, with substantial functional loss, wheelchair dependence and loss of quality of life.

Myotonic Dystrophy Program

Xaira’s internal platforms incorporate leading technologies and personnel which were spun out into Xaira at inception from Illumina’s long-standing functional genomics R&D effort. The company has also integrated a leading proteomics group from Interline Therapeutics. Rodney Lappe, Ph.D., has had a distinguished career as a biopharmaceutical scientist and executive.

Our novel small molecule therapeutic candidates are GeneTAC™ (Gene Targeted Chimera) molecules that target the underlying cause of inherited genetic diseases. Observational Biomarker Study in Friedreich AtaxiaIn parallel to the Phase 1 MAD study, Design conducted an observational study to evaluate FA biomarker assays in blood and skeletal muscle from individuals with FA, FA carriers and normal healthy volunteer controls for use in DT-216 interventional studies. The company developed procedures and methods to measure both FXN mRNA and FXN protein in muscle. Initial data from the observational study is based upon a data cutoff of August 7, 2023. Skeletal muscle biopsies were obtained from study participants at two visits several days apart to measure FXN mRNA and protein levels and characterize inter-and intra-individual variability.

DT-216 muscle exposure in FA patients was lower than projected from animal studies but was sufficient to result in significant PD response in skeletal muscle. Initial data from the Phase 1 MAD trial is based upon a data cutoff of August 7, 2023. The study is fully enrolled but currently ongoing, with blinded PK and pharmacodynamic (PD) data available in the full 100 and 200mg dose cohorts and 11 of 14 participants in the 300mg dose cohort. Ms. Burgess brings more than 25 years of experience in financial reporting, technical accounting, debt and equity financings, and financial planning and management for public and private biotechnology companies.

Program is the Product of a Research Collaboration between Biolojic Design and Nektar entered in 2021

Design modifications offer promise for safer and more effective mRNA therapeutics - News-Medical.Net

Design modifications offer promise for safer and more effective mRNA therapeutics.

Posted: Wed, 06 Dec 2023 08:00:00 GMT [source]

He became involved in Auspex as an investor in 2007 and served as its chairperson until he took over as its chief executive officer in 2013, after which he took the company public and oversaw its growth until its acquisition by Teva Pharmaceuticals for $3.5 billion in 2015. Before that, Dr. Shah was a partner in the healthcare venture capital firm Thomas, McNerney & Partners for nearly a decade, and prior to that, he co-founded two biotechnology companies and was also a consultant at McKinsey & Company in San Francisco. Dr. Shah obtained his Ph.D. in biochemistry and molecular biology as well as his MBA in finance from the University of Chicago. Individuals with nucleotide repeat expansion diseases are born with abnormally expanded stretches of specific nucleotide sequences, often with hundreds to thousands of repeats present in the mutant gene.

DM1 is caused by a mutation in the DMPK gene and is estimated to have a genetic prevalence of 1 in 2,300–8,000 people, affecting more than 70,000 people in the United States and more than 90,000 people in Europe. Each of these programs has the potential to generate blockbuster products with first-in-class or best-in-class profiles. Our proprietary GeneTAC™ platform offers significant potential advantages over other modalities. When systemically administered, GeneTAC™ molecules can distribute widely to reach target cells, overcoming a central challenge for traditional genomic medicines.

The favorable systemic safety profile and FXN response support continued development of DT-216. However, the company has elected to complete dose escalation in this Phase 1 study at the 300mg cohort due to concern for potential worsening of injection site thrombophlebitis at higher doses with multiple administration. Design has shifted focus to developing DT-216 with an improved formulation to enable higher exposures and chronic intravenous administration for treatment of FA. Nonclinical studies showed that the injection site reactions were attributable to the excipients in the current DT-216 formulation, and that improving the formulation composition could enable higher doses and chronic administration.

She currently serves on the grant funding committee for UC Davis and as a charter member for TiE, a non-profit global network of entrepreneurs and venture capitalists. Ms. Prasad earned her bachelor’s degree in business administration at the University of California, Berkeley and her M.B.A. from the Kellogg School of Management at Northwestern University with emphasis in finance and health industry management. Xaira’s advances in biological machine learning are underpinned by the company’s ability to generate, integrate, and learn from vast multidimensional data sets that comprehensively characterize disease-relevant biology at all scales, from molecules to people.

"The board looks forward to supporting the team in building a transformative company under the visionary leadership of Marc Tessier-Lavigne," said director Scott Gottlieb, former Food and Drug Administration commissioner and current NEA partner. "Xaira promises to be a magnet for the best talent in AI and drug development," added director Kaye Foster, Senior Advisor, The Boston Consulting Group and former SVP, Global Human Resources, Onyx Pharmaceuticals. Under the 2021 research agreement, Nektar has tested antibodies computationally designed by Biolojic Design to precisely agonize the TNFR2 receptor. Nucleotide repeat expansion mutations have been identified as the underlying cause of more than 40 debilitating, and typically rare, degenerative genetic diseases that collectively impact millions of people worldwide.

Dr. Xu joined Roche from McKinsey US and worked in biotech R&D earlier in her career. Dr. Xu currently serves on the boards of Walking Fish Therapeutics, Zidan Medical, HBM Healthcare Investments AG and Therorna Inc. She was also previously a board member of ARMO BioSciences (ARMO), NextCure (NXTC), Centrexion Therapeutics and Tempest Therapeutics. We are advancing a pipeline of novel candidates with an initial focus on monogenic repeat expansion disorders with urgent medical need, including Friedreich ataxia, Fuchs endothelial corneal dystrophy, Huntington’s disease and myotonic dystrophy. We are a biopharmaceutical company advancing novel, small molecule therapeutic candidates designed to bring functional cures to serious genetic diseases with long-standing unmet need.

Finally, Jump Financial LLC boosted its position in shares of Design Therapeutics by 284.7% during the third quarter. Jump Financial LLC now owns 46,700 shares of the company's stock worth $110,000 after acquiring an additional 34,561 shares during the last quarter. Pharmacokinetics Plasma PK data were available in participants at 100mg, 200mg, and 300mg DT-216 doses. The average DT-216 concentration in muscle was approximately 8-10nM two days after the third weekly dose and approximately 1nM seven days after the third weekly dose in both the 200mg and 300mg cohorts.

Nucleotide repeat expansions are pathologic when they either impair transcription of the mutated gene (e.g., in Friedreich ataxia) or lead to formation of a pathogenic mRNA or mutant protein (e.g., Fuchs endothelial corneal dystrophy or Huntington disease). A higher number of excess repeats can lead to more severe, and sometimes a more rapidly progressive, form of disease. Over 70% of FECD cases are caused by cytosine-thymine-guanine (CTG) nucleotide repeat expansions in the TCF4 gene, which is transcribed into pathogenic TCF4 RNA that forms nuclear foci and sequesters splicing proteins, leading to transcript mis-splicing (spliceopathy) and CEC death. CECs harbor the longest known TCF4 repeat expansions in the body, potentially explaining why the cornea is the only affected tissue.