Enabling Precision Medicine
Unlocking Oligonucleotide Therapeutics
About Us
At DTx, we are creating novel RNA-based therapeutics to treat the genetic drivers of disease. Our proprietary delivery technology platform utilizes fatty acids as targeting ligands to enable the delivery of oligonucleotide therapies to tissues and cell types throughout the body. In preclinical studies, we have demonstrated cellular uptake and broad activity of oligonucleotides in the retina, muscle, heart, neurons, T cells and many other specialized cell types.
Technology
Historically, this class of medications, which includes siRNA and antisense oligonucleotides has been hampered by challenges with drug delivery. The problem is twofold: inefficient cellular uptake and rapid clearance from the circulation by the kidney and liver.
DTx solves these two fundamental challenges by leveraging decades of knowledge of fatty-acid receptor pharmacology and fatty-acid-based drug delivery technology. The team has developed technology that promotes efficient non-toxic cellular uptake and enhanced systemic bioavailability. We combine the latest in RNA technology with our own proprietary IP to create RNA therapeutics that can efficiently reach targets beyond the liver.
Our approach is analogous to the GalNAc-based conjugation approaches utilized by IONIS, Alnylam, Dicerna and others to enable delivery of RNA medicines to hepatocytes except we enable cellular uptake in many distinct cell types including muscle, heart, immune cells and lung. In addition to universal delivery motifs, DTx is now building a library of cellular uptake motifs that are selective to specific cell types (tropism), potentially expanding the reach of its technology to more specifically target a limited subset of cells.
DTx’s approach is different from lipid nanoparticles (LNPs)—we are tethering RNA medicines directly to naturally occurring fatty acids rather than hiding our drug in a large and highly charged lipid shell. The LNP approach is associated with cytotoxicity and poor biodistribution. The DTx approach has significant advantages related to toxicity, distribution (PK) and manufacturing (PK).
This builds on well-established approaches to improve the half-life and distribution of peptide drugs by conjugation of fatty acids. Albumin serves as an ‘armored Uber’—protecting the RNA drug from circulating nucleases and providing a ‘ride’ via the circulatory system to tissues like muscle, heart and lung. It also limits rapid excretion by the kidney and metabolism by the liver.
It’s worth noting that a fatty-acid based conjugation approach has been successfully deployed to enhance biodistribution of peptide therapeutics—several of which have gone on to become multi-billion dollar drugs including liraglutide, semaglutide, insulin detemir and insulin degludec.
Advantages
DTx believes its approach to delivering RNA medicines solves some of the issues that limited previous-generation platforms, including poor pharmacokinetics and insufficient cellular uptake.
Systemic distribution
DTx motifs drive cargo to muscle, heart and lung and, away from the kidney and liver.
Half-life promotion
DTx technology markedly enhances the residence time of RNA medicines in the circulation, markedly enhancing exposure to tissues beyond the liver.
Cellular
Uptake
DTx motifs enhance cellular uptake via receptor-mediated mechanism at doses equivalent to GalNac efficacy for hepatocytes.
Deep-tissue access
By penetrating tissue layers that virus-based vectors cannot, DTx motifs can be enabling or complementary to other modalities.
Cellular
Tropism
DTx is currently exploring a library of motifs that selectively target specific cell types.
Pipeline
DTx is advancing several internal candidates in addition to partnering on several external programs.
Retinitis Pigmentosa
Retinitis pigmentosa (RP) is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, the light sensitive tissue that lines the back of the eye. Common symptoms include difficulty seeing at night and a loss of side (peripheral) vision. DTx is developing an asset that can address specific genetic subtype of RP called P23H and a separate asset designed to address RP irrespective of the genetic mutation driving disease pathophysiology.
Duchenne muscular dystrophy
DMD is a degenerative disease affecting one in 2,000 boys, whereby a genetic mutation prevents the body from producing dystrophin, a protein that muscles need to work properly. Without dystrophin, muscle cells become damaged and weaken. Duchenne is an irreversible, progressive disease with no cure.
It is known that even if a small amount of functional protein can be made, the disease is significantly ameliorated. Current scientific efforts are focused on creating drugs that allow for production of a truncated, but functional dystrophin protein. The FDA has approved one such drug but its efficacy remains uncertain. Among several reasons, only low levels of the drug reach affected muscle. DTx technology appears well suited to solve this problem in Duchenne, as well as other conditions where exon skipping can be used to produce a functional protein.
Neurodegenerative Diseases of the Central Nervous System (CNS)
Undruggable targets in the CNS cause many of the most serious, debilitating, and life-changing human maladies, including Alzheimer’s, frontotemporal dementia and Huntington’s disease. DTx Pharma has been awarded multiple grants from the NIH (NCATS, NIA), and foundations including the TPEP program of the Alzheimer’s Association and Rainwater Charitable Trust and the CMTRF to advance its efforts to treat CNS diseases.
Management
Arthur T. Suckow PhD
Co-founder and CEOArthur T. Suckow PhD
Dr. Suckow received his BS from the University of Delaware and his PhD from University of California, San Diego. He has worked for Johnson & Johnson, AstraZeneca and Regulus Therapeutics. Dr. Suckow’s extensive experience in fatty acid pharmacology includes advancing small molecules targeting fatty acids and fatty-acid conjugated peptides into early clinical development. Dr. Suckow has received numerous awards including a Beckman Fellowship, NSF graduate research fellowship, a BIOCOM catalyst award and many others.
Charles Allerson
VP ChemistryCharles Allerson
Dr. Charles “Chuck” Allerson joined DTx Pharma as VP of Chemistry and Drug Development in August of 2019. His extensive experience in oligonucleotide chemistry and therapeutics will help accelerate development of the DTx delivery platform and help leverage this technology for the development of new therapies. Prior to joining DTx Pharma, Dr. Allerson served as VP of Chemistry at Stoke Therapeutics, where he supported early stage discovery as well as the development of Stoke’s lead compound, STK-001, for the treatment of Dravet Syndrome.
Bryan Laffitte PhD
Vice President, BiologyBryan Laffitte PhD
Dr. Laffitte brings almost twenty years of pharmaceutical and biotechnology industry experience to DTx, with significant expertise leading research teams across many scientific disciplines and advancing programs from pre-clinical development through IND filings. Dr. Laffitte joins DTx from Inception Therapeutics, Inc., where we served as Vice President, Biology and was responsible for the managing research activities for Inception and several of its subsidiary companies including Tempest Therapeutics, Lycia Therapeutics, Osprey Therapeutics and Metis Therapeutics.
Guriq Basi
CTOGuriq Basi
Dr. Guriq Basi joined DTx Pharma as Chief Technology Officer in February 2020. Dr. Basi has extensive research and management experience spanning start-up biotech to established biopharmaceutical companies, with responsibilities spanning the drug development continuum from target discovery and validation to IND filings. Prior to joining DTx Pharma, Dr. Basi served in several executive level roles including as Principal at GSBio, LLC, CSO, SVP at Sarepta Therapeutics, SVP, Research at Adverum Biotechnologies, CSO at Circuit Therapeutics and Chief Science and Technology Officer at Elan Pharmaceuticals.
Board of Directors
Andrew Adams
CSO, RNA Therapeutics, Eli Lilly & Co.Andrew Adams
Andrew is Eli Lilly’s Chief Scientific Officer for RNA Therapeutics. His eight year tenure at Eli Lilly & Co. includes positions of increasing responsibility in metabolism, diabetes and external innovation. Prior to joining Eli Lilly, Andrew was a Research Fellow at Harvard Medical School. Andrew received his Bsc (honors) and his PhD in Zoology and Neuroscience at the University of Aberdeen in Scotland.
Jeff Friedman
Managing Partner Friedman Bioventure FundJeff Friedman
Dr. Friedman is an experienced life sciences investor with over 40 previous investments in the sector. He is a Pediatric Hematologist/Oncologist, receiving his MD/PhD from Stanford and clinical training at Boston Children’s Hospital/DFCI. Prior to his current role, he led the life sciences section of Tech Coast Angels, San Diego, the largest Angel Investment group in the US. As a philanthropist, Dr. Friedman serves on the board of CurePSP and runs the PSP Genetics Consortium, a collaborative sequencing effort between CurePSP, NIH, the Tau Consortium and 13 academic investigators around the world. Earlier in his career, Dr. Friedman led a research lab for nearly a decade at the Scripps Research Institute in San Diego.
Arthur Suckow
CEO, DTx PharmaArthur Suckow
Dr. Suckow received his PhD from University of California, San Diego and has worked for Johnson & Johnson, AstraZeneca, where he co-founded and managed the biologics laboratory of over 30 scientists, and Regulus Therapeutics. Dr. Suckow’s extensive experience in lipid pharmacology includes advancing lipidated peptides into clinical development and taking small molecules targeting lipid receptors into clinical development.
Tim Scott
CEO, TEGA TherapeuticsTim Scott
Mr. Scott is co-founder and CEO of TEGA Therapeutics. He previously was President of Pharmatek Labs, a CDMO sold to Catalent in 2016. Mr. Scott also serves on the Board of Avelas Bioiosciences, BIOCOM and CONNECT, a non-profit helping to create innovation in San Diego. At UC San Diego, he serves on the Dean’s Advisory Council for the Division of Biological Sciences. Mr. Scott also serves on the board of the San Diego Blood Bank, and as a trustee for the La Jolla Playhouse. Mr. Scott earned his B.A. in Biochemistry, and his J.D. from UCSD and is a member of the California Bar.
John Grundy
Co-founder, DTx PharmaJohn Grundy
Dr. Grundy received his PhD from University of Alberta and has more than 20 years of experience working in the pharmaceutical industry including at Ionis Pharmaceuticals, Eli Lilly & Company, GlaxoWellcome and Elan Pharmaceuticals. He was VP of DMPK & Clinical Pharmacology at Ionis and over his career, has worked on 6 approved therapeutics, including the oligonucleotide therapeutics Kynamro® and Spinraza®, the biologics Tysabri® and Prialt®, and the small molecules Zonegran® and Contrave®. He has proven that he can successfully manage assets from early clinical development through to POC in man. Dr. Grundy is currently VP of Nonclinical Development and Clinical Pharmacology at Arena Pharmaceuticals.
Investors
News & Events
DTx Pharma Announces Presentations at Several Upcoming Virtual Medical Meetings
DTx Pharma, Inc. (DTx), a privately-held biotechnology company creating novel RNA-based therapeutics to treat the genetic drivers of disease, announced today that the company will be presenting at several upcoming [...]
DTx Pharma Announces Appointment of Bryan Laffitte, Ph.D. as Vice President, Biology
DTx Pharma, Inc. (DTx), a privately-held biotechnology company creating novel RNA-based therapeutics to treat the genetic drivers of disease, announced today the appointment of Bryan Laffitte, Ph.D., as Vice [...]