Tackling challenging indications & unmet medical needs.
We are boldly pursuing diseases and conditions where few — if any — effective treatments exist. We embrace the challenge and accept the responsibility to deliver breakthrough options for patients who need them most.
Our Pipeline
Advancing Tr1-based therapies across multiple autoimmune and inflammatory diseases.
Our Programs
We focus on diseases with no effective treatment or cure, prioritizing areas where immune tolerance can transform patient outcomes and redefine standards of care.
Crohn's Disease
Crohn’s Disease (CD) is a type of chronic inflammatory bowel disease (IBD) characterized by abdominal pain, weight loss and significant impact to quality of life. Refractory CD is CD that continues to be inflamed following the failure of two or more approved treatments.
30% of IBD patients fail all lines of therapy
A significant portion of steroid refractory IBD patients never achieve disease control, significantly lowering their quality of life.
D. Wintjens et al. J of Crohn’s and Colitis 15, 391-400(2020)
25% of Crohn’s patients who need surgery
Many Crohn’s patients will ultimately require colectomies to alleviate symptoms, though this does not address the root cause of disease.
Tsai et al. Clin Gastroenterology and Hepatology 19.10 (2021): 2031-2045
Hematopoietic Stem Cell Transplant
Hematopoietic Stem Cell Transplant (HSCT), or bone marrow transplant, replaces a patient’s damaged blood-forming stem cells with healthy ones; it is often the only treatment available for patients with aggressive hematological malignancies, such as myeloid leukemia. Graft versus Host Disease (GvHD) occurs due to pathogenic immune responses after HSCT and can be fatal.
15% rate of severe acute Grade III–IV GvHD
For patients receiving peripheral blood stem cell transplants from mismatched donors.
M. M. Al Malki et al. Blood Advances 5, 2650–2659 (2021).
40% mortality rate for Grade III–IV aGvHD
Despite prophylactic regimens aimed at reducing incidence and severity, over one third of patients with aGvHD die as a result.
Tsai et al. Clin Gastroenterology and Hepatology 19.10 (2021): 2031-2045
Autoimmune Uveitis
Autoimmune Uveitis (AU) is inflammation of the eye’s middle layer (uvea), characterized by symptoms such as redness, pain, light sensitivity and blurry vision. It is caused by the immune system mistakenly attacking its own eye tissues, and is often linked to systemic conditions like lupus or multiple sclerosis. Untreated, AU can lead to vision loss.
Autoimmune uveitis is driven by autoreactive T-cell–mediated inflammation
CD4⁺ T cells disrupt blood-retinal and blood-uveal barriers and recruit inflammatory cells, leading to tissue damage and chronic inflammation.
Papotto, Pedro Henrique, et al. Autoimmunity Reviews, vol. 13, no. 9, 2014, pp. 909–916
Uveitis is a major cause of vision impairment and blindness
It is estimated to account for ~10–15% of blindness in developed countries.
Ghadiri, Nima, et al. BMJ Open Ophthalmology, vol. 8, no. 1, 2023
Progressive Multiple Sclerosis
Progressive Multiple Sclerosis (PMS) is a form of multiple sclerosis characterized by persistent and worsening neurological symptoms like muscle weakness, fatigue and cognitive impairment, leading to eventual loss of ambulatory function and death. While the exact cause of PMS is not known, research suggests that it stems from the immune system mistakenly attacking myelin, the protective sheath around nerve fibers in the brain and spinal cord.
Multiple Sclerosis affects over 2.8 million people worldwide
Onset of symptoms typically begins between 20 and 40 years old, with women 2-3 times more likely to develop MS than men.
Portaccio, Emilio et al. The Lancet Regional Health – Europe, Volume 44, 100977
~15% of patients with MS have Primary Progressive MS
Of the remaining ~85% of patients diagnosed with relapsing remitting MS, most will develop a progressive course over time.
Portaccio, Emilio et al. The Lancet Regional Health – Europe, Volume 44, 100977
Science with purpose
Relentlessly dedicated to improving the lives of patients.
Expanded Access Policy
Our commitment to expanded access
Tr1X is committed to developing products which can substantially improve patient lives by addressing serious or life-threatening diseases with significant unmet medical need. Tr1X’s investigational therapy, TRX319, for the treatment of primary or secondary progressive multiple sclerosis, has received Fast Track Designation from the U.S. Food and Drug Administration (FDA), recognizing its potential to address such need.
This Expanded Access Policy describes how Tr1X evaluates and responds to requests for access to its investigational medicinal products outside of a clinical trial. Such access is permitted under 21 CFR 312.305 and related FDA regulations, is subject to authorization and oversight from regulatory authorities and Institutional Review Boards and may only be considered when certain criteria are met.
Requests for expanded access must be submitted by a licensed treating physician on behalf of a patient and should include the following:
- a summary of the patient’s diagnosis and medical history;
- the rational for requesting access to the investigational medicinal product; and
- an explanation of why alternative therapies and/or clinical trials are not suitable.
These requests should be submitted to the contact information provided below eap@tr1x.bio and should include “Expanded Access Request” in the subject line of the email correspondence. Tr1X will acknowledge receipt of the request within five (5) business days of receiving the request.
Tr1X will evaluate each request for expanded access in MS or other indications in accordance with applicable laws, regulations and company policies. In conducting such evaluation, the following factors may be considered:
- whether the patient has a serious or life-threatening condition and lacks comparable or satisfactory alternative treatment options for their specific situation;
- the rationale for the use of the investigational medicinal product
- potential risk and benefit for the patient in context of the available data; and
- whether providing access would interfere with ongoing or planned clinical trials or drug development.
This Expanded Access Policy is subject to change without notice. Nothing in this Expanded Access Policy shall be construed as a guarantee of access to any investigational product to any individual patient. The investigational therapy described herein has not been approved by the FDA or any other regulatory authority for commercial use. Expanded access use of an investigational product may involve additional risks beyond those involved in participating in a clinical trial.