This “Beta thalassemia - Pipeline Insight, 2020” report provides comprehensive insights about 35+ companies and 35+ pipeline drugs in Beta thalassemia 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
Beta thalassemia Understanding

Beta thalassemia: Overview

Beta thalassemia is a blood disorder that reduces the production of hemoglobin. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body.

In people with beta thalassemia, low levels of hemoglobin lead to a lack of oxygen in many parts of the body. Affected individuals also have a shortage of red blood cells (anemia), which can cause pale skin, weakness, fatigue, and more serious complications. People with beta thalassemia are at an increased risk of developing abnormal blood clots.

Beta thalassemia is classified into two types depending on the severity of symptoms: thalassemia major (also known as Cooley's anemia) and thalassemia intermedia. Of the two types, thalassemia major is more severe.

Symptoms

Children born with Beta thalassemia will have symptoms early in life that include:
Pale skin
Fussy
Having a poor appetite
Having many infections
Over time more symptoms will appear, including:
Slowed growth
Belly (abdominal) swelling
Yellowish skin (jaundice)
Diagnosis

Doctors diagnose thalassemias using blood tests, including a complete blood count (CBC) and special hemoglobin tests.
A CBC measures the amount of hemoglobin and the different kinds of blood cells, such as red blood cells, in a sample of blood. People who have thalassemias have fewer healthy red blood cells and less hemoglobin than normal in their blood. People who have alpha or beta thalassemia trait may have red blood cells that are smaller than normal.
Hemoglobin tests measure the types of hemoglobin in a blood sample. People who have thalassemias have problems with the alpha or beta globin protein chains of hemoglobin.
Treatment

The treatment involves folic acid supplementation in individuals with mild cases of anemia. Supplementation with folic acid, a B vitamin, boosts the production of red blood cells in certain individuals. Along with blood transfusions, folic acid may be given and medications to lower iron levels. In 2019, the Food and Drug Administration (FDA) approved Reblozyl (luspatercept-aamt) for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions. Individuals with beta thalassemia major require regular blood transfusions. A blood transfusion is a common procedure in which affected individuals receive donated blood in order to restore the levels of healthy, functioning hemoglobin to their blood. Some individuals may be treated by the surgical removal of the spleen (splenectomy). An abnormally enlarged spleen (splenomegaly) can cause severe pain and contribute to anemia. Treatment of additional complications of beta thalassemia or iron overload is symptomatic and supportive.

Beta thalassemia Emerging Drugs Chapters

This segment of the Beta thalassemia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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.

Beta thalassemia Emerging Drugs

IMR-687: Imara

IMR-687 is a highly selective and potent small molecule inhibitor of PDE9. PDE9 selectively degrades cyclic guanosine monophosphate (cGMP), an active signaling molecule that plays a role in vascular biology. Lower levels of cGMP are found in people with SCD and beta-thalassemia and are associated with reduced blood flow, increased inflammation, greater cell adhesion and reduced nitric oxide mediated vasodilation. Blocking PDE9 acts to increase cyclic GMP levels, which is associated with reactivation of fetal hemoglobin, or HbF, a natural hemoglobin produced during fetal development. Increased levels of HbF in RBCs have been demonstrated to improve symptomology and substantially lower disease burden in both patients with SCD and patients with β-thalassemia. It is currently in phase II stage of development.

ST-400: Sangamo Therapeutics

ST-400 is being developed by Sangamo Therapeutics for the treatment of Beta Thalassemia. It is currnelty in phase I/II stage of development.

SLN124: Silence Therapeutics

SLN124, which has been shown to lower serum iron levels and modulate tissue iron distribution in rodent models for β-Thalassemia and hereditary hemochromatosis,represents a promising therapeutic candidate for treating patients with iron overload disorders, including β-Thalassemia. It is currently in phase I stage of development.

EDIT-301: Editas Medicine

EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease and β-Thalassemia. EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus. It is in preclinical stage of development.

Beta thalassemia: Therapeutic Assessment

This segment of the report provides insights about the different Beta thalassemia drugs segregated based on following parameters that define the scope of the report.

Major Players in Beta thalassemia

There are approx. 35+ key companies which are developing the therapies for Beta thalassemia. The companies which have their Beta thalassemia drug candidates in the most advanced stage, i.e. phase II include Imara and others.

Phases

This report covers around 35+ products under different phases of clinical development like
Late-stage products (Phase III)
Mid-stage products (Phase II)
Early-stage products (Phase I) along with the details of
Pre-clinical and Discovery stage candidates
Discontinued & Inactive candidates
Route of Administration
Beta thalassemia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Infusion
Intradermal
Intramuscular
Intranasal
Intravenous
Oral
Parenteral
Subcutaneous
Molecule Type
Products have been categorized under various Molecule types such as
Gene therapies
Small molecule
Vaccines
Polymers
Peptides
Monoclonal antibodies
Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Beta thalassemia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Beta thalassemia 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 Beta thalassemia drugs.

Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Beta thalassemia R&D. The therapies under development are focused on novel approaches to treat/improve Beta thalassemia.
In June 2020 Imara announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for IMR-687 for the treatment of patients with beta-thalassaemia.
In January 2019, Silence Therapeutics announces that its lead medicine candidate SLN124 has been granted Orphan Drug Designation by the Committee for Orphan Medicinal Products (COMP), the European Medicines Agency's (EMA).
Sanofi and Sangamo are in partnership for developing gene-edited cell therapies for the treatment of beta thalassemia and sickle cell disease, using Sangamo’s zinc finger nuclease technology.
Beta thalassemia Report Insights
Beta thalassemia Pipeline Analysis
Therapeutic Assessment
Unmet Needs
Impact of Drugs
Beta thalassemia 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 Beta thalassemia drugs?
How many Beta thalassemia drugs are developed by each company?
How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Beta thalassemia?
What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Beta thalassemia 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 Beta thalassemia and their status?
What are the key designations that have been granted to the emerging drugs?
Key Players
Vifor
Sangamo Therapeutics
Acceleron Pharma
Imara, Inc.
Shanghai Bioray Laboratory Inc.
bluebird bio
EmeraMed
Genzyme, a Sanofi Company
La Jolla Pharmaceutical
HemaQuest Pharmaceuticals
Key Products
VIT-2763
ST-400
IMR-687
ACE-536
β-globin restored autologous HSC
GT56-252
LentiGlobin BB305 Drug Product
LJPC-401
HQK-1001