Remarkable progress in the development of cell and gene therapies (CGTx) has ignited hope for millions of patients battling debilitating human diseases including Central Nervous System (CNS) and neuromuscular diseases. However, success of and access to CGTx remain limited due to protocol complexity in small population studies. Jeeva Clinical Trials Inc., a trailblazer in human-centric software solutions to modernize clinical research, offers hope for clinical-stage biopharmaceutical sponsors seeking to execute flexible and complex study designs that are patient-centric, centrally managed, and cost-effective. Innovative biotechnology companies can expect 70% faster study configuration, save researchers between 30%- and 70%-time burden of data collection, and patient engagement through a turnkey solution to unlock the full potential of CGTx.

MANASSAS, Va., July 16, 2024 /PRNewswire-PRWeb/ -- While there are currently hundreds of Cell and Gene Therapies (CGTx) in development worldwide, only about 37 of them have received FDA approval. (1) The emergence of gene therapy represents a breakthrough, particularly in the fight against central nervous system (CNS) and neuromuscular diseases. For the first time in history, it has enabled the possibility to reverse symptoms or substantially slow the progression of certain CNS and neuromuscular disorders. In 2019, the FDA approved Zolgensma, a gene therapy to help stop the progression of a very severe disease called spinal muscular atrophy (SMA) in infants under two years of age. That same year, Jeeva Clinical Trials set out on a mission to modernize clinical research with human-centric software and Contract Research Organization (CRO) solutions.

Research in gene therapy is set to grow exponentially — from a market size of $10.47 billion in 2014 to $52.40 billion by 2033. (2) Even with the approval of Elevidys for Duchenne Muscular Dystrophy (DMD) in June 2024 (3), the promise of CGTx is tempered by major failures of gene therapy programs in the recent years, such as Takeda's gene therapy programs for hemophilia B and Dravet syndrome (4), Biogen's programs for Choroideremia and Retinitis Pigmentosa (5). This, coupled with limited availability, creates a heartbreaking situation for millions who desperately await these life-saving treatments.

While the underlying science for the design and development of CGTx is advancing rapidly, the industry is successfully applying AI-driven drug discovery algorithms to generate drug candidates faster than ever before waiting for clinical evidence generation of safety and efficacy. However, the operational and logistical efficiency gains are yet to catch up to execute over 1000 gene therapy and over 2000 cell therapy clinical trials that are currently active. Most of these qualified CGTx clinical trial sites are in the U.S. and the E.U., while most patients worldwide remain without access.

As Dr. Harsha Rajasimha, CEO and Founder of Jeeva Clinical Trials Inc., notes, "It is very disheartening for a patient or family to know that there is a potential treatment available somewhere, but they can't access it because of where they reside. On the other hand, Biopharmaceutical sponsors are unable to identify enough eligible patients to enroll from within the geographies they operate in. Even when they are successful in enrolling the target number of patients, they are likely missing the genetic diversity or other critical aspects of the disease heterogeneity. Moreover, each genetic disease has a unique distribution of prevalence globally and can disproportionately affect different populations."

Ignoring genetic diversity weakens treatment effectiveness across populations. This has real-world consequences, as exemplified by the Indian diaspora in the US. A study examining 193 rare disease trials found that participants of Indian descent accounted for just 1% of the patient pool. (6) The general dearth of data on Indian patients has led to situations where they received FDA-approved treatments that were ultimately ineffective for their genetic profiles.

Challenges Unique to CGTx clinical trials
CGTx represents a novel and advanced treatment modality with one-and-done administration. Clinical trials for these therapies demand patient-friendly trial designs that fit into their lifestyle with the least burden. These studies tend to involve much smaller patient population compared to other trials involving small molecule drugs or biologics. This means, researchers will need to gather more frequent data points from smaller patient populations including from caregivers and observers of the patients. Most of these patients are severely debilitated due to the disease's nature requiring special needs, choice of communication channels such as video, audio, texting, email, and other special accommodations.

Ensuring long-term patient safety remains challenging for CGTx. The FDA requirement for 15 years of safety follow-up after treatment demands integrated clinical trial management infrastructure including remote patient engagement and patient-centric apps. Additionally, CGTx trials have a need to be more inclusive of non-English speakers and more decentralized due to sparse distribution of the target populations.

Given this set of complex requirements, researchers have relied on CROs selecting 20-30 different software tools and point solutions to make one clinical study successful. Net result is siloed data and fragmented infrastructure resulting in significant integration and interoperability costs.

Patient-Centric Solution to Modernize Genetic Disease Clinical Research
There is good news! Jeeva anticipated the need for human-centric and inclusive clinical research software solutions early on and has developed and validated mature solutions for almost six years now.

Team Jeeva is pioneering patient-centric clinical research software and CRO solutions to lead the way for genetic disease research and CGTx clinical trials at an Enterprise scale, centralizing the management of all studies under a single-login and subscription-based service. "As a rare dad and patient advocate, I realized the only way to address these challenges holistically is with a comprehensive unified solution rather than with piecemeal point solutions that do not integrate and automate the process end-to-end," Rajasimha recounts. And adds, "We cared enough to make time and investments to thoughtfully develop a cost-effective and human-centric solution for patient registries, natural history studies, CGTx clinical trials, and long-term follow-up studies."

Today Jeeva delivers optimized solutions that combines software, experts, and standard processes collectively as a turnkey configurable solution including study specific patient engagement portals. The company's comprehensive clinical research solutions cover software or CRO solutions for protocol design, IRB submissions, site qualification and initiation, patient screening, referral, randomization or enrollment, clinical data management, clinical trial management, patient engagement, biostatistical analysis, and reporting or manuscript preparation for publications. Optimizing all these processes under one-login makes it cost-effective for small population clinical trials or population scale registries or cohort studies.

"Having validated the clinical research software solution for use in settings such as GCP, 21 CFR part 11, HIPAA, with academic medical centers, Children's hospital, and biopharma sponsors, we are partnering with clinical-stage biopharmaceutical sponsors to accelerate the development of affordable CGTx as well as health economics outcomes research (HEOR) studies", Rajasimha highlights.

"With the Diverse and Equitable Participation in Clinical Trials (DEPICT) Act now in play, (7) sponsors of clinical trials must submit a 'diversity action plan' outlining how they will achieve diverse participant enrollment," says Rajasimha. The Jeeva's inclusive clinical research solutions align with the recent FDA guidance to Improve Enrollment of Participants from Underrepresented Populations in Clinical Trials. (8)

Leading Organizations are Choosing Jeeva to Speed up Clinical Research
Modern decentralized and hybrid clinical trials powered by AI are key to unlocking medical breakthroughs. They can boost access and capture richer patient data by making trials remote and inclusive, ensuring diverse representation of patients in their native languages.

COMBINEDBrain, a non-profit organization for rare neurodevelopmental disorders, partnered with Jeeva for this study to capture both quantitative and qualitative data with minimal patient burden. Dr. Terry Jo Bichell, Founder of COMBINEDBrain, states, "We are pleased to collaborate with Jeeva for the eCOA module for our investigator initiator observational study to understand independent toileting abilities by individuals with neurogenetic brain disorders." The study aims to understand the effect of treatments on independent toileting abilities via BYOD surveys with branching logic and scoring, while reducing caregiver and patient burden. Using Jeeva electronic clinical outcomes assessments (eCOA), a software solution that allows researchers to collect, track, and analyze patient and observer reported data, researchers aim to accelerate clinical research programs for various CNS disorders.

CGTx is among the most expensive treatments ever produced, with some exceeding $2 million per dose. (9) We need bold and disruptive models coming from anywhere in the world to ensure access and affordability of CGTx. Jeeva is honored to partner with ImmunoACT to support their vision for making affordable CAR-T Cell therapies. "We needed an innovative platform for our first clinical trial with minimal financial burden and not requiring us to carry a huge IT infrastructure internally. Collaboration with Jeeva was an excellent choice for our CAR-T cell/gene therapy for leukemia and B-cell lymphoma which recently received approval from the Indian regulatory agency for a tenth of the cost compared to the existing CAR-T therapies. This collaboration has been fruitful in storing and sharing clinical data as per regulatory requirements," says Dr. Rahul Purwar, Founder of ImmunoACT and Associate Professor at the Indian Institute of Technology (IIT), Mumbai, India.

Rajasimha and his team were at the ASGCT annual conference earlier this year in Baltimore, MD. Last week, Rajasimha moderated a session on cross-border data sharing for accelerating genetic disease diagnosis and insights at the North America Rare Disease Summit (NARDS) 2024 in Chicago, IL, and will be chairing the Indo US Bridging RARE Summit Nov 16-18, 2024, in New Delhi, India.

Jeeva Pledges Free Software Solutions for N-of-1 Clinical Trials
In a groundbreaking move, Jeeva Clinical Trials Inc. has pledged to provide its software free of charge for all N-of-1 clinical trials like the recently conducted phase 1 trial, AAV gene therapy for hereditary spastic paraplegia type 50 indefinitely. (10) This commitment aims to support personalized medicine and ensure that even the rarest conditions receive the attention they deserve, fostering a more inclusive and effective approach to clinical research.

"This is how we walk the talk of patient-centricity in clinical trials", says an optimistic Rajasimha.

About Jeeva Clinical Trials Inc. ("Jeeva")
Founded by Dr. Harsha Rajasimha after his deeply personal, family, and professional experiences, Jeeva's mission to "Modernize Clinical Research with Human-Centric Software and CRO solutions" propels their innovative clinical trial management platform with AI and workflow automation under one bundled subscription. Jeeva empowers global biopharmaceutical, medical device, and nutraceutical sponsors, simplifying patient engagement and evidence generation, achieving 70% greater efficiency in research. Trusted by prestigious organizations like Frantz Viral Therapeutics and George Mason University, their technology supports diverse study designs, from traditional to fully decentralized models, across disease areas including oncology and rare diseases. With empathy and determination, team Jeeva has developed a patient-centric and unified platform that enables sponsors to conduct clinical trials more effectively, breaking down barriers and fostering a globally inclusive and diverse participant pool. This includes facilitating remote patient participation. Discover more about how Jeeva is reimagining the future of clinical trials at https://jeevatrials.com/.

References
1. "Current FDA Approved Gene & Cell Therapies – Mirus Bio." Mirusbio, 31 May 2024, mirusbio.com/fda-approved-gene-cell-therapies/. Accessed 5 July 2024.
2. "Gene Therapy Market Size Poised to Surge USD 52.40 Billion by 2033." BioSpace, 18 Apr. 2024, biospace.com/article/releases/gene-therapy-market-size-poised-to-surge-usd-52-40-billion-by-2033/.
3. "FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy." FDA, 20 June 2024, fda.gov/news-events/press-announcements/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy.
4. Gardner, Jonathan. "Takeda Drug for Rare Types of Epilepsy Misses Goal in Late-Stage Trial." BioPharma Dive, 24 June 2024, biopharmadive.com/news/takeda-ovid-dravet-lennox-gastaut-failure-soticlestat/719115/.
5. Pagliarulo, Ned. "Biogen Gene Therapy Misses Goal in Eye Disease Study." BioPharma Dive, 14 May 2021, biopharmadive.com/news/biogen-gene-therapy-eye-study/600204/#:~:text=An%20experimental%20gene%20therapy%20did. Accessed 10 July 2024.
6. Chakraborty, Monoswi, et al. "Rare Disease Patients in India Are Rarely Involved in International Orphan Drug Trials." PLOS Global Public Health, vol. 2, no. 8, 15 Aug. 2022, p. e0000890, doi.org/10.1371/journal.pgph.0000890.
7. "Diverse and Equitable Participation in Clinical Trials Act or the DEPICT Act." Congress.gov, Congress of the United States, 3 Feb. 2022, congress.gov/bill/117th-congress/house-bill/6584.
8. "Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies." fda.gov, 3 July 2024, fda.gov/regulatory-information/search-fda-guidance-documents/diversity-action-plans-improve-enrollment-participants-underrepresented-populations-clinical-studies.
9. Stein, Rob. "NPR Choice Page." Npr.org, 2019, npr.org/sections/health-shots/2019/05/24/725404168/at-2-125-million-new-gene-therapy-is-the-most-expensive-drug-ever.
10. Dowling JJ, Pirovolakis T, Devakandan K, Stosic A, Pidsadny M, Nigro E, et al. AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient. Nature Medicine [Internet]. 2024 Jun 28;1–6. Available from: nature.com/articles/s41591-024-03078-4

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