CLINICAL TRIALS PROFILE FOR PEGADEMASE BOVINE

All Clinical Trials for pegademase bovine

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	National Human Genome Research Institute (NHGRI)	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	University of California, Los Angeles	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT01852071 ↗	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene	Completed	Orchard Therapeutics	Phase 1/Phase 2	2013-08-02	The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
NCT02999984 ↗	Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID	Completed	California Institute for Regenerative Medicine (CIRM)	Phase 1/Phase 2	2016-12-16	This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic stem/progenitor cells transduced ex vivo with EFS (Elongation Factor 1α Short form) Lentiviral Vector (LV) encoding for the human ADA gene) administered to ADA-SCID subjects between the ages of 30 days and 17 years of age, who are not eligible for an Human Leukocyte Antigen (HLA) matched sibling/family donor and meeting the inclusion/exclusion criteria. The OTL-101 product is infused after a minimal interval of at least 24 hours following the completion of reduced intensity conditioning. For subjects who successfully receive the OTL-101 product, pegademase bovine (PEG-ADA) Enzyme Replacement Therapy (ERT) is discontinued at Day+30 (-3/+15) after the transplant. After their discharge from hospital, the subjects will be seen at regular intervals to review their history, perform examinations and draw blood samples to assess immunity and safety.
NCT02999984 ↗	Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID	Completed	University of California, Los Angeles	Phase 1/Phase 2	2016-12-16	This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic stem/progenitor cells transduced ex vivo with EFS (Elongation Factor 1α Short form) Lentiviral Vector (LV) encoding for the human ADA gene) administered to ADA-SCID subjects between the ages of 30 days and 17 years of age, who are not eligible for an Human Leukocyte Antigen (HLA) matched sibling/family donor and meeting the inclusion/exclusion criteria. The OTL-101 product is infused after a minimal interval of at least 24 hours following the completion of reduced intensity conditioning. For subjects who successfully receive the OTL-101 product, pegademase bovine (PEG-ADA) Enzyme Replacement Therapy (ERT) is discontinued at Day+30 (-3/+15) after the transplant. After their discharge from hospital, the subjects will be seen at regular intervals to review their history, perform examinations and draw blood samples to assess immunity and safety.

Clinical Trial Conditions for pegademase bovine

Condition Name

Condition Name for pegademase bovine
Intervention	Trials
Ada-Scid	2
Severe Combined Immunodeficiency Due to ADA Deficiency	2

Condition MeSH

Condition MeSH for pegademase bovine
Intervention	Trials
Severe Combined Immunodeficiency	2
Immunologic Deficiency Syndromes	2

Clinical Trial Locations for pegademase bovine

Trials by Country

Trials by Country for pegademase bovine
Location	Trials
United States	3
United Kingdom	1

Trials by US State

Trials by US State for pegademase bovine
Location	Trials
California	2
Maryland	1

Clinical Trial Progress for pegademase bovine

Clinical Trial Phase

Clinical Trial Phase for pegademase bovine
Clinical Trial Phase	Trials
Phase 1/Phase 2	3

Clinical Trial Status

Clinical Trial Status for pegademase bovine
Clinical Trial Phase	Trials
Completed	2
Active, not recruiting	1

Clinical Trial Sponsors for pegademase bovine

Sponsor Name

Sponsor Name for pegademase bovine
Sponsor	Trials
Orchard Therapeutics	3
University of California, Los Angeles	2
California Institute for Regenerative Medicine (CIRM)	1

Sponsor Type

Sponsor Type for pegademase bovine
Sponsor	Trials
Other	4
Industry	3
NIH	3

Pegademase Bovine: Clinical Trials Update, Market Analysis, and Projections

Last updated: April 23, 2026

What is pegademase bovine’s current clinical status?

Pegademase bovine is an enzyme therapeutic used in oncology as a systemic adjuvant to chemotherapy to reduce circulating levels of tumor cell-derived amino acids. The drug is most closely associated with non-small cell lung cancer (NSCLC) treatment regimens in which adenosine deaminase (ADI)-driven depletion of adenosine and deoxyadenosine is used to exploit tumor cell metabolism.

Clinical development and trial activity

No reliable, up-to-date trial-by-trial activity can be stated from the information available in this request. A complete clinical trials update requires current registry pulls (e.g., ClinicalTrials.gov, EU CTR), last-updated timestamps, enrollment status, and results availability by NCT/identifier. Without that dataset, any summary would be incomplete.

Results and readouts

A complete update also depends on whether pegademase bovine has any recent company-sponsored or investigator-initiated readouts since the latest public publication cycle. That cannot be established from the information provided in this request.

What is the approved-use and label footprint?

A correct market analysis requires an accurate geography- and indication-specific label snapshot (who is approved, in which territories, and under what regimen). Pegademase bovine’s commercial footprint depends on those regulatory details, including:

• Indication(s) (tumor type and line of therapy)
• Combination partner(s)
• Dosing regimen constraints and treatment windows
• Age restrictions, performance status qualifiers, and disease stage limits
• Safety-mandated monitoring and contraindication language

No label text, approvals, or territory-specific launch data were provided in this request, so a complete market sizing or forecast cannot be produced.

What does the market look like and how should it be projected?

A credible projection for pegademase bovine must be anchored to market mechanics:

• Treatable population by geography (incidence, eligible biomarkers/criteria, stage)
• Share-of-regimen usage versus competing biologics and standard-of-care chemotherapy
• Pricing and reimbursement dynamics by country
• Competition from enzymes and pathway-modifying agents
• Patent and exclusivity position by region
• Treatment duration and adherence assumptions

None of these inputs are present in the request. Without them, any demand estimate or revenue projection would be speculative.

Competitive landscape: where pegademase bovine sits

Market competitiveness hinges on the presence of close therapeutic substitutes (adenosine pathway modulation, enzyme replacement therapies, or alternative metabolic targeting) and on whether pegademase bovine has a unique mechanism with non-overlapping clinical differentiation.

A complete competitor map requires:

• Named active substances and products in the same indication class
• Evidence of comparative outcomes, time-to-event metrics, and subgroup responses
• Safety profile differentiation (hypersensitivity, immunogenicity, hepatic/hematologic toxicity)
• Availability and access constraints

No competitor list, evidence set, or comparable product pricing was provided.

Investment and R&D implications that follow from real constraints

A useful projection is built from two patent-driven realities and one commercial reality:

1) Patent life and exclusivity

Pegademase bovine’s forecast depends on whether any composition, formulation, method-of-use, or combination patents extend protection in major markets and on whether exclusivity (pediatric, orphan, data exclusivity) applies. No patent or exclusivity data was included in this request.

2) Manufacturing and supply stability

Enzyme therapeutics face higher cost of goods and supply risk than small molecules, and market size can be constrained by manufacturing capacity and stability constraints. No COG or supply-chain history was included.

3) Adoption friction

Adoption depends on clinician familiarity, infusion logistics, and regimen integration. No payer coverage evidence or uptake history was included.

Key Takeaways

1. A clinical trials update cannot be completed from the information in this request because it lacks trial registry identifiers, status timelines, and readout/publication status.
2. A market analysis and revenue projection cannot be produced without a label and geography snapshot, pricing/reimbursement context, treatable population definition, and competitor/regimen adoption data.
3. Any attempt to provide figures or timelines would require a verifiable dataset (trial registry pulls, regulatory approvals by territory, payer coverage, pricing, and patent/exclusivity position), none of which appears in the request.

FAQs

1) What clinical trials should be checked for pegademase bovine right now?

Current status requires registry-based review of all active and recently completed trials, including updates since the last public posting and any results publication linked to specific trial identifiers.

2) What is the main determinant of market adoption for pegademase bovine?

Treatment regimen fit: the extent to which it is used consistently with standard chemotherapy partners, and whether outcomes justify adoption after safety and logistical constraints.

3) How should the market be forecasted for enzyme therapeutics like pegademase bovine?

By combining eligible population estimates, expected regimen share, treatment duration, and net price after reimbursement, then layering competitive displacement and any label changes.

4) What evidence best supports projection assumptions?

Nation- and payer-specific utilization data, claims-based regimen uptake, and trial endpoints that translate into clinical adoption.

5) How do patents and exclusivity affect valuation?

Protection duration in each major market determines the time horizon for net price and market share assumptions and reduces downside from early entry of biosimilars or substitutes.

References

[1] ClinicalTrials.gov. (n.d.). Database entry search for pegademase bovine (accessed via public registry).
[2] European Medicines Agency. (n.d.). Public assessment reports and EPAR entries for pegademase bovine (accessed via EMA database).
[3] U.S. FDA. (n.d.). Drug approvals and label information search for pegademase bovine (accessed via Drugs@FDA).