Table Of Content
Design is pioneering the field of small molecule genomic medicine, which holds the potential to safely and effectively deliver meaningful change to patients in need. Dr. William has served as Managing Partner and Chief Investment Officer of Logos Capital since its founding in 2019. Prior to founding Logos, Dr. Williams served as an investment professional at Farallon Capital Management.
Faculty
The platform is also being built to transform every laboratory and clinical experiment into a vehicle for rich data generation. The product development process itself fuels the development of improved AI models and biological foundation models. The structures of GeneTAC™ molecules enable them to bind and act specifically at the site of the disease-causing nucleotide repeat expansion. They target the mutant gene and modulate the cell’s native transcriptional machinery, selectively halting or resuming the expression of specific genes and in turn blocking pathogenic mRNA or protein production and restoring functional protein production. FA is a multisystem degenerative disease caused by a GAA nucleotide repeat expansion in the FXN gene that impairs transcription and reduces FXN mRNA.
The GeneTAC™ Molecule: A New Class
About Design TherapeuticsDesign Therapeutics is a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC™ gene targeted chimera small molecules. The company’s GeneTAC™ molecules are designed to either dial up or dial down the expression of a specific disease-causing gene to address the underlying cause of disease. In addition to its lead GeneTAC™ small molecule, DT-216, in development for patients with Friedreich ataxia, the company is advancing programs in Fuchs endothelial corneal dystrophy and myotonic dystrophy type-1. Discovery efforts for multiple other serious degenerative disorders caused by nucleotide repeat expansions are also underway, including for fragile X syndrome, spinocerebellar ataxias, Huntington disease, spinobulbar muscular atrophy, and C9orf72-amyotrophic lateral sclerosis/frontotemporal dementia. Design’s lead program is focused on the treatment of Friedreich ataxia, followed by a program in myotonic dystrophy type-1 and discovery efforts for multiple other serious degenerative disorders caused by nucleotide repeat expansions. About Design TherapeuticsDesign Therapeutics is a biotechnology company developing a new class of therapies based on its platform of GeneTAC™ gene targeted chimera small molecules.
Board of Directors
Most recently, Dr. Lappe served as executive chairman of the board of directors for Mirati Therapeutics (MRTX), a role he had served at until he resigned in February 2019, after becoming a member of the board in June 2012 and chairman of the board in July 2013. Previously, Dr. Lappe was group senior vice president, Pfizer Worldwide Research and Development and chief scientific officer for CovX in San Diego. Dr. Lappe has also served as vice president for cardiovascular and metabolic diseases at Pharmacia where he was also site leader for Pharmacia in St. Louis. Prior to joining Pharmacia, he held positions with Wyeth, Rorer Central Research, CIBA Geigy and Searle Pharmaceuticals. "In my previous roles, I witnessed an earlier generation of technologies fundamentally change drug discovery, delivering new medicines that alleviate the burden of disease for many patients," said Dr. Marc Tessier-Lavigne, CEO.
Prior to that, Dr. Jeffries established and led the informatics group at Kalypsys. Dr. Jeffries completed his Ph.D. at the University of Cambridge in epigenetics as an NIH-Cambridge scholar. Sarepta’s gene therapy engine provides a framework for creating a steady stream of new therapies for devastating diseases, such as limb-girdle muscular dystrophies.
Exclusive: In $1B+ bet on AI, biopharma heavyweights back new startup to upend drug R&D
Currently, there are no approved disease modifying therapeutic options that address the cause of nucleotide repeat expansion diseases. Through our proprietary GeneTAC™ approach, we can design and develop therapeutic candidates that dial gene expression up or down without requiring gene editing. HD is caused by a mutation that leads to an increased number of CAG triplet repeats in Exon 1 of the Huntingtin (HTT) gene. Expression of mutant HTT (mtHTT) negatively affects many cellular functions, leading to neuronal death and brain atrophy as symptoms manifest. Wild-type HTT (wtHTT) is thought to be important for normal neuronal function in the adult central nervous system (CNS). It is reported to be involved in axonal transport, synaptic function and cell survival.
Exploring the Potential of Gene Therapies for Limb-girdle Muscular Dystrophies
Design has since shown that an improved formulation had favorable injection site tolerability following multiple intravenous administrations and enabled dosing to increase tissue exposure. Our FECD program leverages our expertise in designing GeneTAC™ small molecules that address the underlying cause of the disease. FECD GeneTAC™ molecules have shown to markedly reduce nuclear foci and improve spliceopathy in FECD CEC cultures derived from donors who underwent corneal transplant.In December 2022, DT-168 was declared a drug candidate for FECD. DT-168 is a GeneTAC™ small molecule designed to target the CTG repeats in the TCF4 gene and selectively block transcription of the expansion-containing allele. He has deep experience developing innovative therapies across multiple diseases and therapeutic modalities through all phases of drug development. Prior to joining Design, Dr. Kim was Chief Medical Officer of Avidity Biosciences.
Design Therapeutics Company Profile
Harnessing synthetic biology for advancing RNA therapeutics and vaccine design npj Systems Biology and Applications - Nature.com
Harnessing synthetic biology for advancing RNA therapeutics and vaccine design npj Systems Biology and Applications.
Posted: Thu, 30 Nov 2023 08:00:00 GMT [source]
Nisa Investment Advisors LLC increased its stake in shares of Design Therapeutics by 38,433.3% in the fourth quarter. Nisa Investment Advisors LLC now owns 11,560 shares of the company's stock worth $31,000 after purchasing an additional 11,530 shares in the last quarter. SG Americas Securities LLC purchased a new stake in shares of Design Therapeutics in the third quarter worth $47,000. Corton Capital Inc. increased its stake in shares of Design Therapeutics by 56.6% in the third quarter.
The variability of results observed with this current method substantially limited the utility of FXN protein measurements to assess DT-216 pharmacology. Myotonic dystrophy (DM1) is a monogenic, autosomal dominant, progressive neuromuscular disease that affects skeletal muscle, heart, brain and other organs. The cardinal features include muscle weakness, myotonia (slow muscle relaxation) and early cataracts. In addition, affected individuals often experience cardiac arrhythmias and changes in neuropsychological function.
There is currently no effective therapeutic intervention that addresses the root cause of the disease. Corneal transplantation, including various modalities of keratoplasty, is the only treatment option to correct advanced FECD. In August 2023, we reported data from the MAD Phase 1 clinical trial showing that DT-216 was generally well-tolerated and exhibited the ability to overcome the FXN transcription impairment that causes FA. We have developed a new drug product candidate, DT-216P2, for patients with FA that demonstrated an improved pharmacokinetic profile and a favorable injection site safety profile in nonclinical studies. Xaira is building significant AI research capabilities spanning fundamental computational methods development and their application to biological discovery, the design of drug-like matter, and clinical development.
Prior to Design, Ms. Burgess served as the senior vice president of finance at Otonomy, Inc., Corporate Controller at Apricus Biosciences, and a consultant to several other biopharmaceutical companies. Ms. Burgess is a licensed CPA and completed her MBA at San Diego State University. This collective knowledge and expertise serve as the engine for our innovative approach and make us uniquely equipped to harness the power of our GeneTAC™ small molecules for the treatment of genetic disease. DT-216 Program Next StepsThe initial results from Design’s Phase 1 multiple ascending dose trial underscore the promise of DT-216 as a potential disease-modifying treatment for FA. FXN protein measurements in skeletal muscle had substantial overlap between FA patients and carriers and showed high intra-individual variability. More than half of FA patients had 25% or more variation in FXN protein levels between visits, with intra-individual coefficient of variation of 69%.
GeneTAC™ molecules are also designed to work with the natural genome without altering a patient’s DNA. Dr. Louise Rodino-Klapac discusses the science of limb-girdle muscular dystrophies (LGMDs) and how our gene therapy engine will help engineer potential therapies for this group of inherited neuromuscular diseases. Royal Bank of Canada restated a "sector perform" rating and set a $4.00 target price on shares of Design Therapeutics in a research report on Wednesday, March 20th. Wedbush restated a "neutral" rating and set a $5.00 target price on shares of Design Therapeutics in a research report on Wednesday, March 20th. Seven analysts have rated the stock with a hold rating, According to MarketBeat.com, the stock currently has a consensus rating of "Hold" and a consensus price target of $5.50.
Affected individuals have reduced life expectancy, with many premature deaths caused by complications of cardiomyopathy at about the end of the fourth decade of life. The estimated prevalence of FA is 1 in 40,000–50,000, affecting more than 5,000 individuals living in the United States and more than 20,000 in Europe. Our team consists of recognized global leaders in the field of DNA-targeted small molecules, transcription modulation, medicinal chemistry, translation, and development into clinical applications. "We have reached the point where AI finally allows us to see biology in new ways, and translate those discoveries to better treatments for disease," said Robert Nelsen, Managing Director and Co-Founder of ARCH Venture Partners. "This creates an enormous opportunity for us to rethink drug discovery entirely. For this reason, Xaira is the largest initial funding commitment in ARCH history."
Deepa Prasad serves as an independent advisor to Équilibre Biopharmaceuticals Corp. and Graviton Biosciences. Prior to vTv, Ms. Prasad was managing director at WestRiver Group, where she led investments in healthcare innovation across biotech and digital health/AI. Ms. Prasad has held various healthcare operational roles as chief of staff at Blue Shield of California, regional vice president for Optum, head of managed care at the California Hospital Association, and vice president, financial strategy and business development at Coherus Biosciences (CHRS). She began her career in investment banking working with biotech and pharma companies on private placements and buyside and sellside mergers and acquisitions, including cross-border M&A with several Japanese pharma.
In addition to its lead GeneTAC™ small molecule, DT-216, in development for patients with Friedreich ataxia, the company is advancing programs in Fuchs endothelial corneal dystrophy, Huntington’s disease and myotonic dystrophy type-1. We are developing a portfolio of GeneTAC™ product candidates designed to address genetic diseases driven by inherited nucleotide repeat expansions. GeneTAC™ molecules are designed to be a novel class of disease-modifying small molecule therapeutic candidates that can either dial up or dial down the expression of a specific disease-causing gene to address the underlying cause of disease. The development of drugs to treat genetic disease has largely focused on novel complex modalities such as gene therapy or gene editing that have proven to be challenging to develop. Until recently, scientists had not succeeded in using small molecules for selective, targeted transcription modulation. Through our GeneTAC™ platform, we have designed small molecules that effectively target and dial up or down the expression of specific genes, with one of our lead compounds having entered the clinic in 2022.
As an exploratory objective, the company also evaluated FXN expression including levels of FXN mRNA and protein in skeletal muscle biopsies obtained at pre-dose baseline and two and seven days after the third weekly dose. In the hands of an experienced team of drug developers, Xaira’s platform forms the basis of a robust therapeutic product development capability across multiple modalities. Its design capabilities, applied to previously difficult to drug targets, are the foundation for a pipeline of differentiated therapeutics.
No comments:
Post a Comment