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.
"It is very disheartening for a patient to
know there is a potential treatment available, but they can't
access it because of where they reside," says Dr. Harsha Rajasimha.
"Biopharmaceutical sponsors also struggle to find eligible
patients, missing genetic diversity."
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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