Hematopoietic Stem Cell Transplantation - Pipeline Insight, 2024

This “Hematopoietic Stem Cell Transplantation - Pipeline Insight, 2024,” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Hematopoietic Stem Cell Transplantation pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Hematopoietic Stem Cell Transplantation: Understanding

Hematopoietic Stem Cell Transplantation: Overview

Bone marrow transplant (hematopoietic stem cell transplant) (HPSCT) involves the administration of healthy hematopoietic stem cells in patients with dysfunctional or depleted bone marrow. This helps to augment bone marrow function and allows, depending on the disease being treated, to either destroy tumor cells with malignancy or to generate functional cells that can replace the dysfunctional ones in cases like immune deficiency syndromes, hemoglobinopathies, and other diseases. The main component of bone marrow which ensures hematopoetic regeneration is the pluripotent stem cell - which has been characterised by immunophenotyping (identification of the cell surface protein/ antigen CD34+ will demarcate cells represent a heterogeneous group including the most primitive blood forming stem cell). Depending upon the donor, the transplantation is called allogeneic, autologous or syngeneic. In each case, the source of these hematopoietic stem cells could be the bone marrow, peripheral blood or umbilical cord blood as mentioned above. In allogeneic bone marrow transplant (allo BMT) the normal hematopoietic stem cells are derived from a HLA matched donor. The donor may or may not be related to the patient (matched sibling donor BMT or matched unrelated donor BMT). In syngenic BMT the donor is an HLA identical twin sibling. In autologous BMT (ABMT) the patient's own stem cells are harvested during remission and used for 50 Years of Cancer Control in India 139 139 hematopoiec rescue after supralethal chemoradiotherapy. The mechanism by which allo BMT is supposed to cure malignancy is by the anti cancer effect of drugs as well as by the immunological reaction between graft and cancer cells known as graft versus leukemia effect (GVL).

Hematopoietic Stem Cell Transplantation- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hematopoietic Stem Cell Transplantation pipeline landscape is provided which includes the disease overview and Hematopoietic Stem Cell Transplantation treatment guidelines. The assessment part of the report embraces, in depth Hematopoietic Stem Cell Transplantation commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence in Hematopoietic Stem Cell Transplantation R&D. The therapies under development are focused on novel approaches to treat/improve in Hematopoietic Stem Cell Transplantation.

• Iomab-B is currently being studied in the pivotal Phase 3 SIERRA (Study of Iomab-B in Elderly Relapsed or Refractory AML) trial, a 150-patient, randomized controlled clinical trial in patients with relapsed or refractory acute myeloid leukemia (AML) who are age 55 and above. Patient enrollment in the SIERRA trial was completed in the third quarter of 2021 and the last patient on SIERRA was transplanted in the fourth quarter of 2021. Topline data results are expected in the third quarter of 2022.

This segment of the Hematopoietic Stem Cell Transplantation report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Hematopoietic Stem Cell Transplantation Emerging Drugs

Iomab-B: Actinium Pharmaceuticals Iomab-B via the monoclonal antibody apamistamab, targets CD45, an antigen widely expressed on all types of blood cancer cells and immune cells including bone marrow progenitor stems cells. Iomab-B links apamistamab to the radioisotope iodine-131 and once attached to its target cells emits energy that travels a relatively long distance, destroying a patient's cancer cells and ablating their bone marrow. By carrying iodine-131 directly to the bone marrow in a targeted manner, Actinium believes Iomab-B will avoid side effects of non-targeted chemotherapy and/or external beam radiation on most healthy tissues while effectively killing the patient's cancer and marrow cells.

Motixafortide: Bio LineRxMotixafortide (formerly known as BL-8040/BKT140) is a novel selective inhibitor of the CXCR4 chemokine receptor. CXCR4, is a well validated therapeutic target that is involved in the mobilization and trafficking of hematopoietic stem cells, immune cells and cancer cells from the bone marrow and the lymph nodes to the peripheral blood. Motixafortide is being developed as a platform for several indications including mobilization of hematopoietic stem cells (HSCs) for autologous transplantations, treatment of solid tumors, and other hematologicalmalignancies.

MANA-312: Mana therapeutics
MANA-312 is an allogeneic donor-derived cell therapy for the treatment of acute myeloid leukemia (AML) in the relapsed refractory post-transplant setting. It is the same product tested in the initial RESOLVE study performed at Children’s National Hospital and Johns Hopkins Medical Center, though enhanced by improvements to the manufacturing process approved by the FDA. The source of the cells is the same donor who provides bone marrow or a leukapheresis for the original bone marrow transplant.

Hematopoietic Stem Cell Transplantation: Therapeutic Assessment

This segment of the report provides insights about the different Hematopoietic Stem Cell Transplantation drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Hematopoietic Stem Cell Transplantation

There are approx. 20+ key companies which are developing the therapies for Hematopoietic Stem Cell Transplantation. The companies which have their Hematopoietic Stem Cell Transplantation drug candidates in the most advanced stage, i.e. Phase III include, Actinium Pharmaceuticals.

Phases

This report covers around 20+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Hematopoietic Stem Cell Transplantation pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intravenous

Molecule Type

Products have been categorized under various Molecule types such as

• Peptide
• Protein
• Propylene glycols
• Cell Therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Hematopoietic Stem Cell Transplantation: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Hematopoietic Stem Cell Transplantation therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Hematopoietic Stem Cell Transplantation drugs.

Hematopoietic Stem Cell Transplantation Report Insights

• Hematopoietic Stem Cell Transplantation Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Hematopoietic Stem Cell Transplantation Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Hematopoietic Stem Cell Transplantation drugs?
• How many Hematopoietic Stem Cell Transplantation drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hematopoietic Stem Cell Transplantation?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Hematopoietic Stem Cell Transplantation therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hematopoietic Stem Cell Transplantation and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Actinium Pharmaceuticals
• BioLineRx
• Athersys
• Novartis
• CareDex
• Orchard Therapeutics
• Magenta Therapeutics
• Graphite Bio
• Vor Biopharma
• Jasper Therapeutics
• Garuda Therapeutics

Key Products

• Iomab-B
• Motixafortide
• HSC Transplant / GvHD
• AlloHeme
• OTL-200
• MGTA 145
• GPH101
• VOR33
• JSP191

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