DTx Pharma2022-10-31T09:16:03-07:00

Breaking open new therapeutic areas for RNA medicines

DTx Pharma is solving one of the biggest challenges limiting the development of genetic medicines: the delivery of RNA therapeutics to tissues beyond the liver.

Our versatile FALCON (Fatty Acid Ligand Conjugated OligoNucleotides) platform conjugates naturally occurring fatty acids to short interfering RNAs (siRNAs) to improve their cellular uptake and bio-distribution. This enables FALCON siRNAs to silence disease-causing genes in tissues beyond the liver.

Fueled by our dual world-class expertise in siRNA design and fatty acid-based delivery, we are using FALCON to expand the target tissues and patient populations that can be served by RNA therapeutics. We have a pipeline of novel therapeutic candidates to treat patients with rare, previously untreatable diseases, including neuromuscular, muscle, and CNS disorders.

Scientific illustration of a guide strand and mRNA being engaged by the RISC.
Scientific illustration of a guide strand and mRNA being engaged by the RISC.

Overcoming obstacles in RNA delivery

siRNAs are a powerful therapeutic modality for treating gene-based disorders with significant advantages over other RNA therapeutic modalities, such as antisense oligonucleotides, including much greater potency, improved safety, and protracted duration of action. However, their advantages have not been fully leveraged to date because they are limited by poor biodistribution and cellular uptake.

DTx’s FALCON technology is overcoming the fundamental challenges associated with siRNA therapeutics by conjugating an siRNA to fatty acids, a tried-and-true mechanism for extending the half-life of multiple, FDA-approved peptide therapeutics. The fatty acid motifs used in FALCON work by both binding to albumin to increase time in circulation to reach target tissues, and leveraging fatty acid receptors on cells to promote efficient cellular uptake once the drug reaches those tissues.

Our groundbreaking, expertise-driven approach is putting a whole new range of target tissues and associated diseases within reach of RNA therapeutics, expanding far beyond the liver to offer life-changing treatment options to many patients for the first time.

Transformative therapeutics for rare disease and beyond.

Our FALCON platform optimizes the siRNA sequence, number of fatty acids, fatty acid type, and linker to safely and effectively deliver siRNA therapeutics to an unprecedented range of organs and cell types. We combine this deep understanding of structure-activity-relationships with world-class siRNA medicinal chemistry capabilities to create highly efficacious therapies tailored to each target tissue. Because we rely on naturally occurring fatty acid motifs that are relatively inexpensive to synthesize, the manufacturing costs are low.

DTx is currently applying FALCON to treat peripheral nervous system, muscular and CNS disorders, with plans to expand to additional therapeutic areas.

Our Lead Programs

Scientific illustration icon of a neuromuscular junction.
Scientific illustration icon of a neuromuscular junction.

Peripheral Nervous System

Our lead program is a first-in-class FALCON siRNA therapeutic called DTx-1252 for Charcot-Marie-Tooth Disease Type 1A (CMT1A). CMT1A is a rare neuromuscular disease that affects 150,000 patients in the US and EU and leads to progressive, severe disability. There are no approved therapies. DTx-1252 targets the repression of PMP22, which is overexpressed in CMT1A patients and is the genetic cause of the disease.

DTx-1252 achieves potent and durable knock down of PMP22 in peripheral nerves and reverses CMT1A in a preclinical rodent model that faithfully recapitulates the genetic and clinical manifestations of the disease. We anticipate moving DTx-1252 into clinical development in 2023.

Muscle

Our FALCON platform permits RNA therapeutics to be delivered to skeletal muscle and we have shown that our FALCON siRNAs can achieve potent and durable knockdown of therapeutic targets across multiple muscle groups. We are making excellent progress optimizing this program and expect to nominate our first development candidate for a muscle disorder in Q4, 2022.

Scientific illustration icon of muscle.
Scientific illustration icon of muscle.

Muscle

Our FALCON platform permits RNA therapeutics to be delivered to skeletal muscle and we have shown that our FALCON siRNAs can achieve potent and durable knockdown of therapeutic targets across multiple muscle groups. We are making excellent progress optimizing this program and expect to nominate our first development candidate for a muscle disorder in Q4, 2022.

Scientific illustration icon of a brain.
Scientific illustration icon of a brain.

CNS

DTx has demonstrated that our FALCON platform enables the delivery of RNA therapeutics to the brain to treat neurodegenerative diseases. We have multiple programs ongoing with data demonstrating preclinical in vivo proof of concept and plan to nominate our first development candidate in 2023.

Other Tissues

DTx is exploring the utility of FALCON in additional tissue types to expand the therapeutic areas we can address with RNA therapeutics, including skin and cardiac tissue. Human primary cell data and in vivo biodistribution studies suggest additional tissues are within reach.

Scientific illustration icon of a heart and skin.
Scientific illustration icon of a heart and skin.

Other Tissues

DTx is exploring the utility of FALCON in additional tissue types to expand the therapeutic areas we can address with RNA therapeutics, including skin and cardiac tissue. Human primary cell data and in vivo biodistribution studies suggest additional tissues are within reach.

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Our Leadership Team

Arthur T. Suckow, PhD

Co-founder and CEO

Arthur T. Suckow, PhD

Dr. Suckow founded DTx Pharma in 2017. An innovative leader in drug discovery, he previously worked on the ophthalmology, diabetes, and NASH programs at Regulus Therapeutics, at the MedImmune arm of AstraZeneca, and in the Diabetes Drug Discovery program at Johnson & Johnson. Dr. Suckow received his BS from the University of Delaware and his PhD from University of California, San Diego. He has received numerous awards including a Beckman Fellowship, a NSF graduate research fellowship, and a BIOCOM catalyst award.

Bryan Laffitte, PhD

Chief Scientific Officer

Bryan Laffitte, PhD

Dr. Laffitte joined DTx Pharma in 2020, bringing twenty years of pharmaceutical and biotechnology industry experience with expertise leading research teams and advancing programs from pre-clinical development through IND filings. Dr. Laffitte was previously Vice President, Biology at Inception Therapeutics where he managed the research activities for Inception and several of its subsidiary companies including Tempest Therapeutics, Lycia Therapeutics, Osprey Therapeutics, and Metis Therapeutics. Dr. Laffitte was previously the Director of Regenerative Medicine, Head of Type 1 Diabetes Program and Co-Leader of the Molecular Targeting Initiative at the Genomics Institute of the Novartis Research Foundation and was named Scientist of the Year and VIVA Award winner for 2016 at Novartis. He completed his postdoctoral fellowship at Howard Hughes Medical Institute; earned his PhD from University of California, Los Angeles; and his BS from California State University, Long Beach.

Charles Allerson, PhD

Senior Vice President of Chemistry

Charles Allerson, PhD

Dr. Charles “Chuck” Allerson joined DTx Pharma in 2019 bringing extensive experience in oligonucleotide chemistry and therapeutics. Prior to joining DTx Pharma, Dr. Allerson served as Vice President of Chemistry at Stoke Therapeutics, Director of Chemistry at Regulus Therapeutics, and Principal Scientist at Isis Pharmaceuticals. Dr Allerson was a postdoctoral research fellow at the National Institutes of Health, completed his PhD at Harvard University, and earned his BS from Lafayette College.

Peter Condon

Chief Business Officer

Peter Condon

Mr. Condon joins DTx Pharma with more than 20 years of experience in business development, commercial planning, and operations. He has held positions as the Vice President and Head of Business Development at Gossamer Bio, and senior roles in business development and commercial planning at Calico Life Sciences, Alexion Pharmaceuticals, and Genentech. Mr.Condon was also responsible for several key corporate transactions across therapeutic areas, including rare diseases, inflammation, and oncology. In addition, he founded an independent consultancy last year where he advised emerging biotech companies on strategy, capital raising, and operations.

Mr. Condon received his BA from Harvard University and his MBA from The Wharton School of Business.

Michael Huang, MD

Chief Medical Officer

Michael Huang, MD

Prior to DTx Pharma, Dr. Huang held key leadership roles at AmMax Bio and Spruce Biosciences. Throughout his career, he has led programs in RNA therapeutics, biologics and small molecules from initial IND through clinical proof-of-concept, late-stage development and beyond. With extensive leadership experience in advancing drug candidates through all phases of clinical development, his contributions have led to marketing authorizations for numerous therapeutics including Potiga®, Zevalin®, Uceris®, Ruconest®, and Austedo®.  Dr. Huang has also been recognized for supporting successful financing and business-development activities.  He is also the author of a multitude of peer-reviewed journal articles, abstracts, and scientific publications.

Dr. Huang holds a Bachelor of Science in molecular and cell biology from the University of California at Berkeley and received his medical degree from the Chicago Medical School.

Our Board of Directors

Arthur Suckow, PhD

CEO, DTx Pharma

Arthur 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.

Matthew Hammond, PhD

Board Member

Matthew Hammond, PhD

Matthew Hammond joined RA Capital in 2014 and is currently a Principal on the Investment Team. Matthew works on both public and private investments and serves as a Director for Cerebral Therapeutics, Kira Pharmaceuticals, and Research Alliance Corp I. Matthew holds a BS in Environmental Health Science from the University of Georgia and both a PhD in Biomedical Science and an MBA with a concentration in Finance from the University of Connecticut. His graduate research investigated innate immune responses after intracerebral hemorrhage.

Dan Becker, MD, PhD

Access Biotechnology

Dan Becker, MD, PhD

Dan Becker is a Partner at Access Biotechnology, the biopharmaceutical investing arm of Access Industries, a privately held US-based industrial group with global strategic investments of $25+ billion. Prior to joining Access, Dan was a Principal at New Leaf Venture Partners, where he played a key role in multiple private and public biopharma investments, and a Principal in the Health Care practice at the Boston Consulting Group, where he led projects across the healthcare sector with an emphasis on biopharma R&D. Dan trained clinically in internal medicine and nephrology at Brigham and Women’s Hospital and Massachusetts General Hospital, and was a Research Fellow at Harvard Medical School. He obtained both his MD and PhD (Cellular and Molecular Biology) degrees from the University of Michigan, and received his BS in Physiology from the University of Illinois at Urbana-Champaign.

Kathie Bishop, PhD

Independent Board Member

Kathie Bishop, PhD

Dr. Bishop brings more than two decade’s worth of experience in translational research and drug development, with a focus on novel therapeutics for the treatment of neurological and rare diseases. She is currently Sr. Vice President, Chief Scientific Officer and Head of Rare Disease at Acadia Pharmaceuticals. As an accomplished scientist-turned-executive, Dr. Bishop has previously led research and development as CSO at Locana Bio, Otonomy and Tioga Pharmaceuticals and held leadership roles at Ionis and Celgene.

Jeff Friedman, MD, PhD

Managing Partner, Friedman Bioventure Fund

Jeff Friedman, MD, PhD

Dr. Friedman is an experienced life sciences investor with over 50 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 served on the board of CurePSP and continues to manage 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 basic hematology research lab for nearly a decade at the Scripps Research Institute in San Diego. In addition to DTx Pharma, Dr. Friedman is also on the board of Pet Dx.

Tim Scott

CEO, TEGA Therapeutics

Tim 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, PhD

Co-founder, DTx Pharma

John Grundy, PhD

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.

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Careers

At DTx, we are a group of driven, rigorous and innovative thinkers who are determined to solve one of the biggest challenges limiting the development of genetic medicines on behalf of patients everywhere.

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