Views and missives from the frontier

March 14, 2022

CAMP4 Therapeutics Appoints Veteran Drug Developer Ann Barbier, M.D., Ph.D. as Chief Medical Officer

  • Al Sandrock, M.D., Ph.D. joins Scientific Advisory Board

CAMBRIDGE, Mass., March 14, 2022 – CAMP4 Therapeutics, a biotechnology company harnessing the power of regulatory RNAs to upregulate genes and restore healthy expression, today announced that it has appointed Ann Barbier, M.D., Ph.D., as Chief Medical Officer, responsible for leading all clinical research and development, medical affairs and related functions. Additionally, former Biogen Executive Vice President of R&D, Al Sandrock, M.D., Ph.D., will join CAMP4’s scientific advisory board.

“Ann and Al bring exceptional and complementary expertise to CAMP4 as we prepare for our transition to a clinical-stage company,” said Josh Mandel-Brehm, CEO of CAMP4. “Ann is a proven leader and drug developer who has successfully brought multiple rare disease medicines through clinical development. Al is one of the foremost drug developers in neurology and a long-time champion of CAMP4; we are delighted to have him join us in a more formal capacity as we advance our lead program in Dravet syndrome through IND-enabling studies this year.”

Prior to joining CAMP4, Dr. Barbier was Chief Medical Officer of Translate Bio, leading the development of an inhaled mRNA for the treatment of cystic fibrosis. Her prior experience includes positions at Agios Pharmaceuticals, Shire, EnVivo Pharmaceuticals, Johnson & Johnson and Aventis. During her career, Dr. Barbier has made significant contributions to several approved products such as Elaprase® (Hunter syndrome), Aubagio® (multiple sclerosis), Firazyr® (hereditary angioedema) and Pyrukynd® (pyruvate kinase deficiency) and has led several investigational new drug applications for new chemical entities. She is a member of the Board of Directors of Pieris Pharmaceuticals. Dr. Barbier received her M.D. and a Ph.D. in pharmacology from Ghent University, Belgium, and a Master of Science from the Free University of Brussels, Belgium. She pursued a postdoctoral fellowship at the University of Tennessee in Memphis.

“The pharmaceutical industry has really begun to appreciate the potential of RNA as a target for therapeutic development and CAMP4’s focus on regulatory RNA is the next frontier,” said Dr. Barbier. “I am excited to take the reins of the near-term clinical programs, expand the pipeline into additional genetic diseases and join the executive team as we guide the company towards becoming a clinical-stage organization.”

Dr. Sandrock joins CAMP4’s scientific advisory board following a prolific career in biopharmaceutical drug development. Dr. Sandrock spent 23 years at Biogen where he identified and developed novel therapies for a variety of serious neurological diseases, including Tysabri®, Tecfidera®, Spinraza®, Plegridy® and Aduhelm™. While at Biogen, he served in positions of increasing responsibility, culminating in his service as Executive Vice President, Research & Development. He also held a seat on the Biogen Executive Committee as Chief Medical Officer.

“CAMP4 is leveraging a proven technology coupled with a powerful discovery platform to create new potentially disease-modifying medicines for patients with genetic disorders for which, right now, symptom management is the best we can offer,” said Dr. Sandrock. “Having watched the company’s science mature in recent years, I believe CAMP4 is capable of exploiting a vast opportunity to enable tunable upregulation of gene expression to treat disease and I look forward to lending my experience to help guide these efforts.”

CAMP4 combines its proprietary RNA Actuating Platform (RAP) with state-of-the-art oligonucleotide technology to develop RNA Actuators, precise and programmable therapeutics that uniquely upregulate gene expression at the transcriptional level. CAMP4’s approach targets a new class of RNA known as regulatory RNAs (“regRNAs”) that control the expression of mRNA, making this approach applicable to any genetic disease whereby a small increase in gene output can lead to meaningful therapeutic outcomes. CAMP4’s current pipeline is focused on diseases where oligonucleotides have proven safe and effective, including CNS and liver diseases, with the potential to expand to a broad array of tissues.

CAMP4 has multiple RNA Actuator programs in pre-clinical development in CNS and liver diseases. The company’s candidate molecule for Dravet syndrome, a form of severe genetic epilepsy, is designed to directly upregulate endogenous SCN1A gene expression by targeting Natural Antisense Transcripts, a subset of regRNAs that control mRNA transcription. CAMP4 expects to file an IND for this program by the end of 2022. CAMP4 is also developing a molecule for urea cycle disorders (UCD), a set of debilitating and life-threatening liver diseases, the most common form of which is linked to loss-of-function mutations in the ornithine transcarbamylase (OTC) gene. CAMP4’s RNA Actuator aims to upregulate endogenous production of enzymes of the urea cycle in order to improve the patient’s ability to process and eliminate excessive ammonia that causes the cognitive delays, comas, and seizures that are the hallmarks of UCD.

About CAMP4 Therapeutics

At CAMP4, we are pioneering a novel approach to programmable therapeutics. We combine a deep understanding of regulatory RNA and gene expression with a complementary and customizable oligonucleotide modality. Our RNA Actuating Platform’s proprietary insights enable us to harness the power of RNA to upregulate the expression of genes and unlock the potential to create treatments for hundreds of diseases affecting millions of patients. Learn more about us at and follow us @CAMP4tx.


Lindy Devereux
Scient PR