Category Archives: Cell Therapy

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People with lupus know theres no cure for their condition. Treatment options usually include medications to prevent flare-ups and manage symptoms, but nothing to effectively control the disease progression.

However, researchers at UC Davis Health want to change that, as they are striving to improve the quality of life for people living with lupus through a novel clinical trial evaluating CAR T-cell therapy as a treatment for the autoimmune disease.

Lupus, formally known as Systemic lupus erythematosus (SLE), is a chronic autoimmune disorder that occurs when the bodys own defenses, the immune system, attacks the body by mistake. In this case, the cells of the immune system that produce antibodies called B cells attack healthy tissue. This leads to inflammation and damage to multiple organs, including the skin, joints, kidneys, heart and lungs.

Another subtype of the condition is lupus nephritis (LN), when lupus affects the kidney. LN causes progressive damage to the kidneys and can lead to kidney failure if not properly treated.

Lupus can be treated with immunosuppressive medications, but the therapy options are limited with variable efficacy and disease control, explained Gaurav Gulati, medical director of Rheumatology, Allergy and Clinical Immunology and principal investigator of the study. There is no cure for lupus, so these drugs are taken over prolonged periods of time, which can cause additional side effects. There is a significant need for targeted and more effective treatment options for individuals with lupus. This is vital for improving long-term control of this disease and also improve patient outcomes.

About 1.5 million Americans, and at least five million people worldwide, have a form of lupus according to the Lupus Foundation of America. Anyone can develop lupus, but certain people are at higher risk, including:

Modified T cells, known as CAR T cells, are an FDA-approved treatment for different forms of cancer including acute lymphoblastic leukemia, non-Hodgkin lymphoma, and multiple myeloma. With cancer, the immune system often fails to deploy T cells right away or at all. When it does, the attack is ineffective. CAR T-cell immunotherapy changes these collected T cells to produce chimeric antigen receptors (or CARs) that adhere to tumors to destroy them.

A recent study published in the New England Journal of Medicine, suggests CAR T-cell therapy could become a highly effective treatment for SLE patients who do not respond to current lupus therapeutics.

The study included 15 patients eight with lupus, four with systemic sclerosis (scleroderma), and three with idiopathic inflammatory myositis, a rare muscle disease. Researchers were able to eliminate or reduce symptoms and disease biomarkers with a single infusion of CAR T cells designed to target B cells, the immune cells that are key to driving autoimmunity.

There were no relapses among the studys lupus patients, who were monitored for up to two years after the treatment.

The new, industry-funded clinical trial with Cabaletta Bio is evaluating CABA-201, an investigational therapy in patients with either SLE or LN who have active disease. UC Davis Health is one of just nine health care institutions worldwide and the only hospital in the Western United States to participate in the clinical trial.

CABA-201 is a chimeric antigen receptor CAR T-cell therapy. Participants in the study will have their blood drawn and filtered through a machine in a process called apheresis. The procedure separates part of the blood such as platelets or white blood cells, while returning the rest of the blood to the body.

The trial will isolate T cells in the blood and researchers will genetically modify, or change, the T cells by putting in a code to add the CAR. This way, they can recognize, attack, and destroy the B cells in the participants body. This includes the bad B cells that engage in the participants autoimmune disease.

Once the T-cells are modified, or changed, they are supported to increase in number to produce a dose of CABA-201 cells. The CABA-201 cells are infused into the participants body, where they attack the B cells, eliminating the bad B cells and potentially enabling healthy B cells to repopulate in the body.

Our primary goal for this phase of the trial is to evaluate the safety and effectiveness of the single dose infusion of the CAR T-cell therapy, noted Gulati. If successful, this one-time treatment will correct the underlying defect and improve the quality of life for these patients.

Additional UC Davis sub-investigators for the clinical trial include Mehrdad Abedi, professor of internal medicine in the division of hematology and oncology, and rheumatology fellows Stacey Guo and Ruchi Shah. The team aims to enroll 12 patients between the ages of 18 and 65, six of them with active LN and six with active non-renal SLE.

For more information about this trial, contact the UC Davis study team at (916) 237-8850 or the sponsors study team can be reached at (267) 759-3100 or clinicaltrials@cabalettabio.com.

You can also take a survey on UC Davis StudyPages to learn if you may qualify for enrollment.

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A breakthrough for lupus treatment? Study explores CAR T-cell therapy for autoimmune disease - UC Davis Health

A 27-year-old woman with hard-to-treat systemic lupus erythematosus (SLE), the most common type of lupus, became the first patient treated in an early clinical trial evaluating FT819, Fate Therapeutics off-the-shelf CAR T-cell therapy candidate.

The Phase 1 study (NCT06308978), taking place at the University of Minnesota Medical School and the University of Nebraska Medical Center, aims to assess FT819s safety, pharmacological properties, and initial activity in as many as 32 adults with moderate to severe SLE.

In what Fate called a first-of-kind experiment, researchers collected blood samples from the woman diagnosed more than 10 years earlier with SLE before treatment was administered. They then added FT819 to those samples and waited 24 hours to see the effects the cell therapy might have.

T819 was found to rapidly eliminate B-cells, which are immune cells that drive lupus by producing antibodies, called autoantibodies, that wrongly target healthy tissues.

Three days after treatment, the patient was discharged from the hospital without any notable adverse events, according to acompany press release.

With CAR T-cell therapies, immune T-cells isolated from patients or healthy donors are modified to carry a lab-made chimeric antigen receptor (CAR) that directs them to attack a specific target. FT819s CAR targets CD19, a protein found on the surface of B-cells. The therapy is designed to deplete B-cells, lower the levels of autoantibodies, control lupus symptoms, and prevent organ damage.

Before receiving a one-time infusion of the cell therapy, patients must undergo a lymphodepletion regimen with chemotherapy agents to eliminate disease-driving immune cells.

A CAR T-cell approach targeting CD19 was recently validated in an academic investigator-sponsored study, which showed that all patients with lupus-related kidney complications achieved drug-free, durable remission.

The seminal data with autologous CAR-T cell therapy demonstrating early and long-lasting remissions in patients with certain B cell-mediated autoimmune diseases is remarkable, and we are very excited to bring potentially novel therapeutic solutions with disease-modifying potential to our patients, said Jennifer Medlin, MD, the trials principal investigator at the University of Nebraska.

Unlike some other CAR-T cell therapies that require a patients own T-cells known as an autologous treatment Fate uses a proprietary method to create CAR T-cells from induced pluripotent stem cells (iPSCs). These iPSCs are a type of stem cells that can be created by reprogramming different types of adult cells back to a state in which they can give rise to any type of cell in the body.

According to the company, iPSC-derived CAR T-cells are uniform in composition and can be stored for so-called off-the-shelf use, potentially reaching more patients.

These solutions may extend to off-the-shelf cell products, such as FT819, which may have the potential to overcome critical challenges that could limit patient access to CAR-T for autoimmune diseases, Medlin said, citing conditioning chemotherapy and extended hospitalization, among other factors.

Fate presented recent data supporting the use of FT819 in lupus at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, held May 7-11 in Baltimore.

In preclinical studies using blood from lupus patients, FT819 rapidly depleted CD19-positive B-cells in a dose-dependent manner.

The company presented translational data from a Phase 1 study of FT819 in people with hard-to-treat cancers affecting B-cells, or B-cell malignancies (BCM). These data highlighted key therapeutic mechanisms of FT819 for treating autoimmune diseases driven by B-cells.

From blood samples collected from 23 treated BCM patients, FT819 led to the rapid and sustained depletion of CD19-positive B-cells. This was followed by the recovery of nave and immature healthy B-cells, with little or no recovery of B-cells that produce disease-driving autoantibodies. The therapy also eliminated CD19-positive B-cells in tissues, including bone marrow, lymphoid tissue, and the liver.

We are excited to bring our iPSC product platform and our first product candidates to patients with autoimmune diseases, where preclinical and translational data from our off-the-shelf FT819 CAR T-cell program demonstrate key therapeutic mechanisms of activity for autoimmunity.

Fate also is developing FT522, a similar CD19-targeted, off-the-shelf CAR therapy using immune natural killer (NK) cells instead of T-cells, for patients with B-cell lymphoma, a type of blood cell cancer. A novel re-challenge assay using blood from lupus patients showed that FT522 rapidly depleted CD19-positive B-cells. Similar results were found using blood from the first two lymphoma patients from a Phase 1 study,

We are excited to bring our iPSC product platform and our first product candidates to patients with autoimmune diseases, where preclinical and translational data from our off-the-shelf FT819 CAR T-cell program and our FT522 CAR NK cell program demonstrate key therapeutic mechanisms of activity for autoimmunity, said Scott Wolchko, president and CEO of Fate.

We believe these programs have a favorable safety profile, offer patient access and convenience, and can deliver the breadth and depth of B cell depletion necessary to induce immune reset in patients with B-cell mediated autoimmune diseases, Wolchko added.

Continued here:
1st lupus patient treated with CAR T-cell therapy FT819 in early trial - Lupus News Today

Galapagos has signed a strategic partnership with Blood Centers of America (BCA) to expedite the decentralised production of its chimeric antigen receptor (CAR)-T cell therapies across the US.

This strategic partnership will utilise BCAs extensive network, spanning over 50 community blood centres in 43 states.

The deal will facilitate in the good manufacturing practice (GMP)-compliant manufacturing of Galapagos CAR-T haemato-oncology clinical programme.

Using BCAs network, Galapagos will conduct technology transfers to various sites concurrently with the decentralised production of the CAR-T product candidates close to cancer treatment centres.

Galapagos will also access apheresis capacity at BCA sites when required.

BCA will play a vital role in coordinating site initiation and onboarding processes, which will help Galapagos speed up its manufacturing efforts and maintain consistent quality across all locations.

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Galapagos decentralised manufacturing platform is designed to deliver fresh, fit cells with a median vein-to-vein time of just seven days.

Galapagos CEO Dr Paul Stoffelssaid: Our collaboration with Blood Centers of America marks a major milestone for Galapagos US expansion by establishing centres for support of our pivotal studies, with the potential to be used for commercial introduction.

Working with BCAs extensive nationwide network allows us to efficiently scale up decentralised CAR-T therapy manufacturing across the US, while their established infrastructure helps us harmonise operations and access apheresis capacity.

This agreement complements the existing collaborations between Galapagos and Landmark Bio and Thermo Fisher Scientific and translates to a significant step forward in our aspiration to bring CAR-T therapies to more patients with a vein-to-vein time of just seven days.

The latest development comes after Galapagos and BridGene Biosciences entered a strategic partnership and licensing deal for the discovery of new small-molecule drug candidates for oncology targets.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported byCytiva.

Editorialcontent is independently produced and follows thehigheststandardsof journalistic integrity. Topic sponsors are not involved in the creation ofeditorialcontent.

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Galapagos enters deal to boost CAR-T therapy production - Pharmaceutical Technology

James Ignatz-Hoover, MD, PhD, hematologist/oncologist, University Hospitals Seidman Cancer Center, discusses the sequencing of CAR T-cell therapy and bispecific antibodies in the treatment of patients with relapsed/refractory multiple myeloma.

Ignatz-Hoover notes that both CAR T-cell therapies, ciltacabtagene autoleucel (cilta-cel; Carvykti) and idecabtagene vicleucel (ide-cel; Abecma), have demonstrated deep responses in patients with relapsed/refractory multiple myeloma. Both agents were previously approved by the FDA for the treatment of patients with relapsed/refractory multiple myeloma following 4 or more prior lines of therapy, including a proteasome inhibitor (PI), an immunomodulatory drug (IMiD), and an anti-CD38 monoclonal antibody. Notably, the BCMA-directed bispecific antibodies teclistamab-cqyv (Tecvayli) and elranatamab-bcmm (Elrexfio) are both approved in the same indication.

Notably, on April 5, 2024, the FDA expanded the indication for cilta-cel to include the treatment of adult patients with relapsed/refractory multiple myeloma who have received at least 1 prior line of therapy, including a PI and an IMiD, and who are refractory to lenalidomide (Revlimid). On the same day, the FDA expanded the indication of ide-cel to include the treatment of adult patients with relapsed/refractory multiple myeloma after 2 or more prior lines of therapy, including an IMiD, PI, and anti-CD38 monoclonal antibody.

When treating a patient in a setting where a CAR T-cell therapy and a bispecific antibody are both approved, Ignatz-Hoover explains that CAR T-cell therapy requires an extended vein-to-vein time to manufacture the product. Development is underway to address the manufacturing time with next-generation CAR T-cell therapies using rapid protocols in an effort reduce vein-to-vein time, he says.

In comparison to CAR T-cell therapy, bispecific antibodies provide an off-the-shelf treatment option for patients who cannot afford to wait for weeks for CAR T-cell manufacturing, Ignatz-Hoover continues. He explains that bispecific antibodies may also be better tolerated and offer a viable alternative for patients who may not tolerate CAR T-cell therapy.

Ignatz-Hoover emphasizes that these treatment modalities exist along a spectrum of options, catering to the diverse needs of older and frailer patients with multiple myeloma.

The sequencing of CAR T-cell therapy and bispecific antibodies is a critical aspect of treatment decision-making for heavily pretreated patients with multiple myeloma, and Ignatz-Hoover concludes by saying that a tailored approach considering patient characteristics and treatment goals is essential in optimizing outcomes in heavily pretreated patients.

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Dr Ignatz-Hoover on Sequencing CAR T-Cell Therapy and Bispecific Antibodies in R/R Myeloma - OncLive

Patients with lymphoma have gained several treatment options with the wave of CAR T-cell therapy approvals by the FDA in recent years. However, additional work is required to find better methods to overcome mechanisms of resistance to these agents, according to Sattva S. Neelapu,MD.

Most recently, on May 15, the FDA granted accelerated approval to the CAR T-cell agent lisocabtagene maraleucel (liso-cel; Breyanzi) for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) who have received 2 or more prior lines of systemic therapy. The regulatory decision was supported by findings from the phase 2 TRANSCEND-FLstudy (NCT04245839). Findings from the study demonstrated that patients who received liso-cel (n = 94) experienced an overall response rate of 95.7% (95% CI, 89.5%-98.8%). Additionally, the median duration of response was not reached (NR; 95% CI, 18.04-NR) at a median follow-up of 16.8 months (95% CI, 16.3-17.0).

TRANSCENDFL showed very high efficacy in [the] third-line [setting], Neelapu said. [These findings] led to the approval of liso-cel in [the] third line.

In an interview with OncLive, Neelapu, the deputy department chair and a professor in the Department of Lymphoma/Myeloma, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center in Houston, provided an overview of the currently available CAR T-cell therapies in FL, mantle cell lymphoma (MCL), and large B-cell lymphoma (LBCL), as well as the mechanisms of resistance patients can develop to these treatments and how they could potentially be addressed.

Neelapu: There are 4 different CD19-[directed] CAR T products that are currently approved [by the FDA]. With respect to LBCL, initially the approvals were in the third-line setting and that was based on 3 single-arm, [early] phase studies: ZUMA-1 [NCT02348216], JULIET [NCT02445248], and TRANSCEND-NHL-001 [NCT02631044]. Those [studies] showed very high efficacy, with [approximately] 40% of patients achieving durable remissions.

Then, CAR T-cell therapy was evaluated in the second-line setting in randomized phase 3 trials. At least 2 of the trials were positive and led to FDA approvals in the second-line setting, including ZUMA-7 [NCT03391466] testing axicabtagene ciloleucel [axi-cel; Yescarta] and the TRANSFORM study [NCT03575351] testing liso-cel. Both studies showed that CAR T-cell therapy improved event-free survival as well as overall survival in the case of the ZUMA-7 study compared with the existing standard of care, which is salvage chemotherapy followed by stem cell transplantation. This created a paradigm shift in how we treat patients with LBCL in the second-line setting; CD19-[directed] CAR T-cell therapy is the new standard of care.

There is also another trial, the [phase 2] TRANSCEND-PILOT-017006 study [NCT03483103] that evaluated liso-cel in transplant-ineligible patients in the second-line setting. This was a single-arm study that also showed very high efficacy and tolerability, with approximately 40% of patients achieving durable remission. Now both transplant-eligible as well as transplant-ineligible patients with LBCL have CD19-directed CAR T-cell therapy as the new standard of care in second-line setting.

In the FL space, there are 2 trials that have shown high efficacy in the third-line setting; these are single-arm, phase 2 studies. Theres ZUMA-5 [NCT03105336] which is testing axi-cel and the ELARA trial [NCT03568461] which is testing tisagenlecleucel [tisa-cel; Kymriah]. Both studies showed a complete response rate in the 70% to 80% range and showed very high durability. For example, in the ZUMA-5 study, at 4 years of follow-up, more than 50% of patients were still progression free. Both of these trials led to the FDA approvals of axi-cel and tisa-cel in the third-line setting of follicular lymphoma.

In the MCL space, the initial trial that led to an approval was the ZUMA-2 study [NCT02601313] evaluating brexucabtagene autoleucel [brexu-cel; Tecartus]. That was a single-arm study of patients who have had both alkylating therapy, anti-CD20 agents, and BTK inhibitor therapy. That trial showed an approximate 70% complete response rate with ongoing remissions in approximately 35% to 40% of patients at 3 years. Theres also the [MCL cohort of the] TRANSCEND study, which evaluated liso-cel in MCL. That also showed very high efficacy but is not yet FDA approved, although it is also expected to lead to an FDA approval later this month in MCL.

One of the main reasons that patients relapse after CD19-[directed] CAR T-cell therapy is [due to] loss of CD19 in the tumor at progression. That can be addressed by developing CAR T-cell therapy against other targets in B-cell lymphoma, including CD22, CD79, and others. [There was a phase 1/1b clinical trial examining] CD22-directed CAR T that was done at Stanford University [NCT04088890] that is now in phase 2 clinical trials. There are also other trials that are currently in early stages of development.

The second major reason that patients relapse after CAR T-cell therapy is because of T-cell dysfunction. The T cells in these patients are dysfunctional at the time of apheresis, and that yields a poor-quality CAR T product. The T cells become dysfunctional either because of the disease itself, or more commonly because of various therapies that the patients have received.

One way to address that is to move CAR T-cell therapy to earlier lines [of treatment]. Another way to address that is to alter the manufacturing process. Traditionally, CAR T-cell production used to take 7 to 10 days, at least for the currently approved commercial products. But recent data indicate that if we reduce the manufacturing time to 2 to 3 days, it yields a much better-quality CAR T product that has better function in patients and better efficacy without an increase in toxicity. A third way to overcome the T-cell dysfunction issue is to develop allogeneic CAR T-cell therapies. There are a number of different allogeneic CAR T-cell therapy approaches that are currently being evaluated in phase 1 and 2 trials.

FDA grants accelerated approval to lisocabtagene maraleucel for follicular lymphoma. FDA. May 15, 2024. Accessed May 16, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-lisocabtagene-maraleucel-follicular-lymphoma

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CAR T-Cell Agents Offer Efficacy in Lymphoma, But Additional Approaches Are Needed to Overcome Resistance - OncLive

Bristol Myers Squibb was granted accelerated approval from the FDA for Breyanzi(lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy, for the treatment of adults with relapsed or refractory follicular lymphoma (FL) who have received two or more prior lines of systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent on verification and description of clinical benefit in confirmatory trial(s). In relapsed or refractory FL, Breyanzi is delivered as a one-time infusion with a single dose containing 90 to 110 x 106 CAR-positive viable T cells. The approval includes Boxed WARNINGS for Breyanzi regarding Cytokine Release Syndrome (CRS), Neurologic Toxicities, and Secondary Hematological Malignancies. Historically, FL has been considered an incurable disease, and patients frequently relapse following front-line therapy, with prognosis worsening after each subsequent relapse. Despite advances in treatment, there remains an unmet need for additional options that offer treatment-free intervals with durable, complete responses. The Phase 2 TRANSCEND FL study included the largest primary analysis set of patients with relapsed or refractory FL of a clinical trial evaluating a CAR T cell therapy in this patient population. In patients treated with Breyanzi in the third-line plus setting, the overall response rate (ORR) was 95.7%, as defined as the percentage of patients achieving a partial or complete response per an Independent Review Committee. The complete response (CR) rate was 73.4% and required a negative bone marrow biopsy for confirmation. Responses were rapid and durable with a median time to response of one month and median duration of response (DOR) not reached, with 80.9% of responders remaining in response at 12 months, and 77.1% of responders remaining in response at 18 months. Breyanzi has exhibited a consistent safety profile and across clinical trials, any grade cytokine release syndrome (CRS) occurred in 53% of patients, including Grade >3 CRS in 4% of patients. The median time to onset was 5 days. Any grade neurologic events (NEs) occurred in 31% of patients, with Grade >3 NEs occurring in 10% of patients. The median time to onset of NEs was 8 days. The safety profile of Breyanzi allows for the option of outpatient treatment and management of patients.

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BMS's CAR T Cell Therapy Breyanzi Approved for Follicular Lymphoma - Contract Pharma

Editor's Note: May 28 is World Blood Cancer Day

ROCHESTER, Minn. Chemotherapy is usually the first treatment doctors try to treat lymphoma, including the two most common forms: non-Hodgkin and Hodgkin. But alternatives to chemotherapy are developing, as first-line treatments and as backup options, explains Stephen Ansell, M.D., Ph.D., hematology chair and hematologic oncologist at the Mayo Clinic Comprehensive Cancer Center.

Lymphoma is a blood cancer that begins when a germ-fighting white blood cell, called a lymphocyte, mutates and rapidly multiplies. There were roughly a half-million new cases of non-Hodgkin lymphoma and 82,409 new cases of Hodgkin lymphoma in 2022, making them the 10th and 26th most commonly diagnosed cancers that year, respectively, according to the most recent statistics from the World Health Organization's International Agency for Research on Cancer.

Symptoms may include swollen lymph nodes, itchy skin, night sweats, fever, persistent fatigue, unexplained weight loss and shortness of breath.

Developing novel therapies to improve lymphoma outcomes for patients is a high priority for Dr. Ansell.

"It's about improving outcomes while minimizing side effects using treatments that can specifically target the cancer and have less of an impact on the body's healthy, normal cells," Dr. Ansell says. "There is a high percentage of patients whose lymphoma is cured. When people are cured, they may have to deal with long-term complications. Our idea is to have the best outcomes and minimize long-term problems."

Dr. Ansell's studies have included work as part of Mayo's Early Cancer Therapeutics Group. The therapeutics research group offers patients whose cancers haven't responded to chemotherapy the opportunity to join early phase clinical trials of potential new treatments.

People with lymphoma may receive chemotherapy alone or a combination of chemotherapy and nonchemotherapy treatments, Dr. Ansell says. Nonchemotherapy options include immunotherapy, chimeric antigen receptor-T cell therapy (CAR-T cell therapy), targeted therapy, bone marrow transplant and radiation therapy, he explains:

Unfortunately, there is no known way to prevent lymphoma, but a healthy diet and exercise are important. They can help patients better cope with cancer treatments and have better outcomes, Dr. Ansell says.

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Fighting lymphoma: Treatment options include alternatives to chemotherapy, expert explains - Mayo Clinic

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BioSolution Designs and RoosterBio Announce Collaboration on Genetically Modified MSCs for Biotherapeutic Development BioBuzz has been building life science communities for 14 years because we believe in the power of personal networks to unlock your career potential. For our next chapter, were building upon the success of our community to help solve todays greatest challenges in hiring and career development. There is a better, more equitable way to connect talent with opportunity and were building it.

Frederick, MD, May 2, 2024 BioSolution Designs (BSD), a biotechnology invention studio developing proprietary platforms for multigenic gene and cell therapies, today announced its ongoing strategic collaboration with RoosterBio, a leading provider of human mesenchymal stem/stromal cells (MSCs), highly engineered media solutions, and bioprocess development services.

This partnership leverages BSDs multigenic design and assembly platform, Bird of Prey, and cell engineering expertise along with RoosterBios cutting-edge MSC and exosome bioprocessing products, manufacturing protocols, and analytical services to simplify the development and manufacturing of engineered MSC derived therapies.

Together, BSD and RoosterBio deliver an end-to-end solution for the engineering and development of cell- and exosome-based therapeutics, including BSDs genetic engineering expertise and RoosterBios critical raw materials, expert process development, and advanced analytical characterization. Both teams have proven capabilities to deliver engineered therapies that possess a favorable global regulatory profile.

Dr. Thomas D. Reed, CEO of BSD, commented on the partnership, saying, Mesenchymal stem cells represent a major untapped resource with broad therapeutic utility, from regenerative medicine to treating rare diseases. Understanding all too well that The Product is the Process, we are thrilled to collaborate with RoosterBio, whoseraison detrerevolves around controlling all aspects of scalable cGMP manufacturing and characterization of human MSCs and MSC-derived exosomes. This partnership will not only benefit our internal therapeutic initiatives but will provide a seamless transition for BSDs OspreyBio customers to send multigenic therapeutic candidates to RoosterBio for efficient manufacturing.

BSD is at the leading edge of advanced therapy engineering, said Tim Kelly, CEO of RoosterBio. By combining Biosolution Designs innovative multigenic technologies with our well-established cell therapy products and scalable bioprocess manufacturing platforms, we will accelerate the clinical progression of the next generation of cell and exosome therapeutics.

For further information, please visit:

BioSolution Designs:www.biodzn.com

RoosterBio:www.roosterbio.com

OspreyBio:www.ospreybio.com

BioSolution Designs (BSD) is a biotechnology invention studio developing first-in-class multigenic therapies for complex diseases using proprietary purpose-built multigenic technology platforms to create, control, deliver, and manufacture such therapies for cardiovascular, neurodegenerative, and musculoskeletal diseases. BSD invents multigenic platforms and commercializes them as biotools through its wholly-owned OspreyBio operations. BSD also leverages the platforms to develop (up through IND filing) multigenic therapeutics and spin them out into disease-area-focused therapeutic companies. BSDs platform/biotools plus its therapeutics spinout business model enables distribution of external investments pursuant to specific investor interests in the platform/biotools business or the therapeutics spinouts, as each have different risks and return profiles. The Founder and CEO of BSD is Dr. Thomas Reed, who was previously the Founder and Chief Science Officer of Intrexon, now known as Precigen.

RoosterBio accelerates human mesenchymal stem/stromal cell (hMSC) and extracellular vesicle (EV) product and process development to fuel the rapid implementation of scalable advanced therapies. Our high-quality hMSCs, bioprocess media, genetic engineering tools, and EV production solutions are paired with expert bioprocessing knowledge to progress therapeutic developers from concept to first-in-human testing and commercial manufacturing at reduced cost and increased productivity. With optimized, scalable processes, Type II Master Files, and cGMP products, we have enabled therapeutic programs to traverse their path to clinical translation in under one year. RoosterBio is driven by clients success and creating a world where safe and effective regenerative medicines are rapidly developed and widely available on a global scale.

OspreyBio, a wholly owned subsidiary of BSD and a leading provider of reagents and tools that simplify the development and delivery of complex DNA and RNA therapeutics. The company, led by CEO Sam Glickstein, was founded in 2022 and is headquartered in Frederick, MD. OspreyBio is the only company dedicated to making multigenic research the new standard for complex therapy R&D. Todays multigenic diseases pose the biggest unresolved threats to human health. However, there are no accessible, easy-to-use multigene vector construction tools or multigene delivery tools on the market. The gene and cell therapy industry is still relatively nascent and genetic engineering technologies available to date have limited current approved and many therapies in the clinic to monogenic rare diseases. OspreyBio seeks to introduce novel multigenic gene and cell therapy biotools to enable gene and cell therapy research on complex multigenic diseases, such as heart disease, Alzheimers, and many solid tumors.

BioBuzz is a community led, experience focused, biotech and life sciences media and events company. BioBuzz highlights regional breaking news, industry professionals, jobs, events, and resources for business and career growth. Their weekly newsletter is subscribed to by thousands in the BioHealth Capital Region and Greater Philadelphia as the go-to for industry updates.

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BioSolution Designs and RoosterBio Announce Collaboration on Genetically Modified MSCs for Biotherapeutic ... - BioBuzz

People with chronic inflammatory diseases are taking part in a new cell therapy clinical trial that one participant said made them feel miles better.

The POLARISE trial, being organised by the University of Birmingham and funded by a grant from Innovate UK is testing a type of cell therapy called Stromal Cells to see whether they can resolve symptoms and inflammation in patients with certain autoimmune diseases including Rheumatoid arthritis and Primary Sclerosing Cholangitis.

POLARISE is a Phase 2 trial which will investigate the safety and activity of ORBCEL a stromal cell therapy that has been developed by Orbsen Therapeutics Ltd. Stromal cells are rare cells found naturally in the human body where they stimulate resolution of injury and inflammation via a natural healing process called Efferocytosis. Stromal cells are also allogeneic which means they can be purified from one donor and given to multiple patients without causing allergic reactions - so there is no need for donor matching.

These rare stromal cells are ethically sourced and purified from human donor tissue and expanded to therapeutic doses at the University of Birmingham Medicines Manufacturing Facility (MMF).

The ORBCEL therapy is administered intravenously across two visits with subsequent hospital appointments to check on the progress of their condition during a two-year trial period.

Philip Newsome, Professor of Hepatology and Honorary Consultant Hepatologist at the University of Birmingham is leading the POLARISE trial. Professor Newsome said: Stromal cells are an exciting potential treatment for inflammatory diseases. These diseases are debilitating and very hard to treat as the body has switched a natural defence system for dealing with threats to one that starts attacking itself. Its therefore critical to find ways to support the body to naturally deal with inflammation rather than turn off the defences which can lead to all sorts of infections.

Early results from previous trials using Orbsens ORBCEL stromal cell therapy are encouraging and were hopeful that the treatment will be beneficial for some patients.

Early results from previous trials using Orbsens ORBCEL stromal cell therapy are encouraging and were hopeful that the treatment will be beneficial for some patients.

Hannah Dines, Rio 2016 Paralympian: Im still going strong

Self-confessed type A person, Hannah Dines is one for setting mad goals. Born with cerebral palsy, freelance writer and sportswoman Hannah trained and raced for Great Britain in para-cycling including racing at the Rio 2016 Paralympic games, and now represents GB in adaptive surfing.

However, in 2021 during the buildup to the delayed Tokyo games Hannah was diagnosed with a chronic inflammatory disease called Primary Sclerosing Cholangitis (PSC) in which the bile ducts in the liver get progressively narrower can lead to liver failure and impacts other organs like the spleen, intestines and bowel.

Hannah explains: I was diagnosed with PSC after struggling with major fatigue and worsening of my spasticity from my cerebral palsy. I would train and feel very ill but do it anyway. I love moving my body and during the training I still felt that joy. Still, I began to fear the symptoms that would come after. I used to call it having an exercise hangover though I rarely drank alcohol and was in my twenties. I would ensure I had at least four hours after training to collapse in bed, too tired to even watch TV, feeling too ill to sleep, known as malaise.

By the point of diagnosis though I was really ill and sleepy every day, I couldnt focus but I kept pushing with my training, a part time job and then bed. Finally, a clinical doctor took my blood to put me on an alternative spasticity medication that required a liver function test. Thats when I was sent to a liver clinic and they took a liver biopsy right away and found out my sclerosis was pretty serious.

It made all my symptoms make sense and because I was young and sporty no-one misdiagnosed me with fatty liver or alcoholism, which was nice, even if it didnt really lessen the impact of having PSC.

After receiving her diagnosis, qualified physiologist Hannah knew she wanted to try and take part in a clinical trial although received a series of rejections due to the advanced nature of her PSC.

Hannah continues: I was recommended for POLARISE and I didnt hesitate. The day after my first dose I felt incredible and not just because the clinicians administering the drugs were so nice. This effect lasted a couple of days and I truly felt released from PSC.

I was still competing at a sport: adaptive surfing and I booked all my contests because I knew I wouldnt need to cancel. I laughed out loud on an aeroplane because I felt real energy for the first time in years. It was probably the steroids or a placebo effect but my liver function tests also got much better.

My second dose was a little underwhelming compared to my first, but I still felt miles better. My malaise and feeling kind of dead had gone away.

I used to obsess over my blood values and stopped checking them. I started setting goals more than two months in advance, which I had decided not to do after a year of having to cancel everything. Were now six months and I still big hits of malaise but just to know that respite might be possible like at the start of my trial...that's really special..

All I can do is hope the findings are positive and this can become a regular treatment for people with PSC. No matter the result of POLARISE it has given me real hope for the future.

Not letting PSC stop her, Hannah has taken up adaptive surfing and last year represented GB at the world championships, finishing fourth in her category and supporting Team GB to their most successful championships yet.

Stromal Cells

Stromal cells such as Orbsens ORBCEL therapy can be purified from bone marrow or umbilical cord tissues donated by healthy individuals with donor consent under ethical approval by the Anthony Nolan Trust. While each single bone marrow or umbilical cord contains only few thousand stromal cells these cells can be purified by Orbsens technology to undergo controlled expansion in cleanroom bio-reactors to produce a thousand allogenic doses of ORBCEL from each tissue.

Within the Innovate UK-funded Advanced Therapies Treatment Centre (ATTC) Consortium and POLARISE trial - these tissues are transported from the Anthony Nolan centres to the Advanced Therapies Facility (ATF) at the University of Birmingham - where Orbsen and ATF staff collaborated to purify and manufacture doses of Orbsens Stromal cell therapy ORBCEL- using patented technologies and Terumos Quantum Cell Expansion Bioreactors.

Orbsen Therapeutics Chief Scientific Officer, Steve Elliman said: We are delighted to continue our significant and productive clinical collaborations with Prof. Newsome, the University of Birmingham - and the Anthony Nolan Trust - to determine the safety and efficacy of our ORBCEL therapies in patients with chronic inflammatory diseases.

These First in Human (FIH) trials are difficult to undertake and deliver even more so during the COVID19 pandemic. These trials are not possible without brave patients - like Hannah Dines - who volunteer to participate in these rigorous safety trials. And so, we take this opportunity to thank the patients, nurses and clinical teams who work so hard to complete these invaluable studies.

We look forward to completing these important safety trials and look forward to examine how ORBCEL can encourage resolution of symptoms in patients with chronic inflammatory disease.

The Innovate UK-funded POLARISE trial represent the third major clinical trial collaboration between The University of Birmingham and Orbsen Therapeutics to assess the safety and efficacy of Orbsens ORBCEL therapy. Professor Phil Newsome is also leading the EU FP7 funded MERLIN clinical trial that is assessing ORBCEL as a therapy for patients with autoimmune liver diseases. The MERLIN trial is complete and is expected to report in the first half of 2024.

Orbsen is also collaborating with Prof Paul Cockwell at the University of Birmingham and Professor Giuseppe Remuzzi at the Mario Negri to assess the safety of ORBCEL as a therapy for Chronic Kidney Disease caused by Type 2 diabetes, in a Phase 1/2 clinical trial called NEPHSTROM. Professors Cockwell and Remuzzi recently published the first results from NEPHSTROM in the prestigious Journal of the American Society of Nephrology (JASN). In the NEPHSTROM trial publication in JASN, a low dose of ORBCEL was reported to be safe and promote stabilization of kidney function over 18 months in patients suffering with Progressive Chronic Kidney Disease and type 2 diabetes.

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New stromal cell treatment for chronic inflammatory diseases being trialled - University of Birmingham