Aruvant Chooses Lonza to Manufacture ARU-1801, a Potentially Curative Treatment for Sickle Cell Disease, for Pivotal Trial
January 18, 2021

NEW YORK, Jan. 18, 2021 /PRNewswire/ -- Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant's one-time investigational gene therapy for sickle cell disease (SCD). Aruvant has chosen Lonza to help develop and manufacture ARU-1801 for its upcoming pivotal trial.

"We are partnering with Lonza, one of the world's leading cell and gene therapy manufacturing organizations, to help us manufacture our gene therapy ARU-1801, a potential cure for sickle cell disease that can be given with one low dose of chemotherapy," said Palani Palaniappan, Aruvant chief technology officer. "Our internal cell therapy process expertise combined with Lonza's cell processing know-how provide the perfect combination to manufacture ARU-1801 for our pivotal study. As we advance our gene therapy through clinical trials, this partnership signifies a critical milestone in the development pathway."

Aruvant is building a hybrid supply model that is led by the company's cell and gene therapy scientists and leverages specialized external suppliers. The company has expanded the internal technical and operational organization with experts in vector and cell manufacturing, analytics, quality, supply and regulatory affairs. To complement the internal capabilities, Aruvant is partnering with world-leading contract development manufacturing organizations. The strategic long-term manufacturing agreement with Lonza is a critical step to advancing the clinical development of ARU-1801 for sickle cell patients.

"Our track record in the cell and gene therapy space makes us the ideal partner to help manufacture ARU-1801, a differentiated gene therapy that is an important future option for patients with sickle cell disease," said Alberto Santagostino, senior vice president, head of cell and gene technology, Lonza. "Through a strong integrated team effort, together we can bring a life-changing medicine to patients."

Under Aruvant's direction, Lonza has begun process development and technology transfer activities from its Houston center of excellence to help establish robust manufacturing for ARU-1801. Once complete, Lonza will deliver cGMP material supply for the pivotal clinical trial.

About ARU-1801
ARU-1801 is designed to address the limitations of current curative treatment options, such as low donor availability and the risk of graft-versus-host disease (GvHD) seen with allogeneic stem cell transplants. Unlike investigational gene therapies and gene editing approaches which require fully myeloablative conditioning, the unique characteristics of ARU-1801 allow it to be given with reduced intensity conditioning ("RIC"). Compared to myeloablative approaches, the lower dose chemotherapy regimen underlying RIC has the potential to reduce not only hospital length of stay, but also the risk of short- and long-term adverse events such as infection and infertility.

Aruvant is conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time potentially curative investigational gene therapy for patients with SCD. This Phase 1/2 study is currently enrolling participants, and information may be found at  which includes a patient brochure, an eligibility questionnaire and information for healthcare providers.

About Aruvant Sciences
Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an active research program with a lead product candidate, ARU-1801, in development for individuals suffering from sickle cell disease (SCD). ARU-1801, an investigational lentiviral gene therapy, is being studied in a Phase 1/2 clinical trial, the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. For more information on the clinical study, please visit and for more on the company, please visit Follow Aruvant on Twitter @AruvantSciences and on Instagram @Aruvant_Sciences.

About Roivant
Roivant's mission is to improve the delivery of healthcare to patients by treating every inefficiency as an opportunity. Roivant develops transformative medicines faster by building technologies and developing talent in creative ways, leveraging the Roivant platform to launch Vants – nimble and focused biopharmaceutical and health technology companies. For more information, please visit

About Lonza
Lonza combines technological innovation with world class manufacturing and process excellence. Together, these enable Lonza's customers to deliver their discoveries in the healthcare, preservation, and protection sectors.

Lonza is a preferred global partner to the pharmaceutical, biotech and specialty ingredients markets. Lonza works to prevent illness and promote a healthier world by enabling customers to deliver innovative medicines that help treat or even cure a wide range of diseases. In addition, Lonza offers a broad range of microbial control solutions, which help to create and maintain a healthy environment.

Founded in 1897 in the Swiss Alps, Lonza today operates in 120 sites and offices in more than 35 countries. With approximately 15,500 full-time employees, Lonza is built from high-performing teams and of individual employees who make a meaningful difference to the business, as well as the communities in which the company operates. The company generated sales of CHF 5.9 billion in 2019 with a CORE EBITDA of CHF 1.6 billion. Find out more at and follow Lonza on Twitter @LonzaGroup or Facebook @LonzaGroupAG.

SOURCE Aruvant Sciences

For further information: Aruvant Contact: E. Blair Clark-Schoeb, Senior Vice President, Communications,; Lonza Contacts: Dr. Sanna Fowler, Tel +41 61 316 8929,; or Dirk Oehlers, Lonza Group Ltd,