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Patient-centric drug development 

Capital-efficient drug development is key to offsetting the spiralling costs of discovering drugs and bringing new therapies to patients. Pricing pressure has forced new strategies to evolve with many biotech companies typically involved in the early stages of drug discovery embracing a new paradigm of patient centricity and focus that brings the importance of patient advocacy to the fore of clinical trial design, drug development and ultimately drug access. 


Now more than ever, companies developing therapeutics are seeking deep engagement with patient communities earlier in the drug development process. This, in turn, leads to improvements in clinical trial enrollment, better informed, more robust, and relevant outcomes from trials, shorter drug development timelines, increased likelihood of successful regulatory engagement, approval and drug launch, payor drug coverage and reimbursement, and broad uptake of marketed products by the prescribing and clinical community.

Patient communities are playing increasingly active roles in clinical trial protocol co-creation. Working with patients and  disseminating clinical trial information in a patient-friendly manner is now a top priority for many companies. 

The ability to engage with patient communities and to share their voices with regulatory agencies, payors, and health care providers is an essential part of the drug development journey. It is particularly important in order to gain rapid translation of critically needed orphan drug products for diseases with high unmet clinical need. Patient advocacy — from basic research through clinical trials to approved and reimbursed therapy — is critical. The ability to present the patient perspective to help speed regulatory approval and articulate patient benefit versus the traditional lens of economic argument for limitations around drug coverage and patient access is paramount to ensure that new treatments ultimately reach patient communities without delay. 


RENOGENYX is committed to deeply understanding the lives of patients living with FSHD and the impact of this disease on those who care for them. Patient experiences and the unique challenges they face every day from the lack of accurate, affordable, and accessible diagnostics, to misdiagnosis and misunderstanding from the medical community, to the uncertainty of living with a randomly progressive degenerative muscular dystrophy are what drive us to develop a cure.

Our scientists are global FSHD key opinion leaders who have been at the forefront of academic research. Our team is responsible for many of the scientific breakthroughs and progress made in the field, including understanding the underlying mechanisms of disease, the development of animal models to validate therapeutic candidates, and the development of appropriate diagnostics.  

Our mission, with help and guidance from the patient community, is to develop safe and effective treatments for FSHD as quickly as possible. 

If you, your loved one, or family are affected by FSHD and want to find out more about CRISPR inhibition therapy and our drug development program, please get in touch by emailing us at

This website may contain general information relating to various medical conditions and their treatment.

Such information is provided for informational purposes only and is not meant to be a substitute for advice provided by a doctor or other qualified health care professional. Patients should not use the information contained herein for diagnosing a health problem or disease. Patients should always consult with a doctor or other health care professional for medical advice or information about diagnosis and treatment.

CRISPRi Gene Therapy for FSHD, or any other therapeutic program referenced on this website, is not an approved therapeutic product. The information on this website is for illustrative purposes only.

Any suggested treatment protocol, diagnostic process, or any other information related to any medical product is entirely proposed only. No suggestion is made as to the efficacy or safety of any therapeutic product in development. Any approved therapeutic product and/or treatment regimen for any therapy being developed by RENOGENYX may be materially different in scope, nature, and practice than any suggested.

No reliance should be made upon any information presented in this website as being fit for any purpose. Please see the Terms of Use for this website for details of forward-looking statements & risk which apply to any drug development program described, suggested, or referenced, in whole or part on this website. 

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