Skip to main content

Beam Therapeutics (Nasdaq: BEAM) Portfolio, Stock and Growth

 Highlights of Beam Therapeutics 

- Biotechnology company developing genetic medicine 
- Use proprietary CRISPR based editing technology
- Portfolio includes CAR-T cells for leukemia
- Exvivo editing for sickle cell disease
- In vivo editing for Wilson Disease
- Cash or Cash equivalents  $202.2 million 

Beam Therapeutics, launched in 2018, is a biotechnology company developing genetic medicine through the use of CRISPR base editing technology. Beam’s proprietary base editors create precise, predictable, and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that The company is using to advance a diversified portfolio of base editing programs. The company is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases 

Beam Therapeutics (Nasdaq: BEAM) 


Platform and Delivery System

The novel base editing approach uses a chemical reaction designed to create precise, predictable, and efficient genetic outcomes at the targeted DNA sequence. By rewriting a single base in the genome, base editors enable a diverse toolkit that could therapeutically intervene to correct disease-causing point mutations, modify genes to create protective genetic variations, activate gene expression, silence gene expression, or “multiplex” and make multiple simultaneous edits.

The clinically validated technologies are used for delivery to these genomic medicines. They are 
  • electroporation,
  • non-viral and
  • viral delivery modalities

Beam Therapeutics Platform & Therapeutic Areas

Sickle Cell Disease and Beta Thalassemia
Acute Myeloid and Lymphoblastic Leukemia
Liver Diseases
Ocular Diseases






Base Clinical/Research Program Update

BEAM-201 for Treatment of T-ALL 
Beam is advancing BEAM-201, a potent and specific anti-CD7 CAR-T multiplex editing program for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL), a severe disease affecting children and adults with five-year overall survival of less than 25%.
BEAM-201 is produced using a GMP-compliant, clinical-scale process in which T-cells derived from healthy donors are simultaneously base edited at four genomic loci then transduced with a lentivirus coding for an anti-CD7 CAR. The resulting cells are universally-compatible, allogeneic (“off the shelf”) CD7-targeting CAR-T cells resistant to both fratricide and immunosuppression. 

Glycogen Storage Disease Type Ia 
Beam’s approach to treating patients with GSDIa is to deliver an ABE via lipid nanoparticle (LNP) to the liver to repair either the R83C or the Q347X mutations in G6PC. It is estimated that these two-point mutations account for 900 and 500 patients, respectively, in the United States, representing approximately 60% of all GSDIa patients.

In vivo correction of both mutations by ABEs was observed in the livers of two strains of transgenic mice, each carrying one of the two G6PC mutations. Next-generation sequencing data from whole liver extracts reveal significant correction for both R83C and Q347X, with nearly 40% and approximately 70% A-to-G conversion efficiency, respectively, of each mutation back to the normal gene sequence. These significant levels of mutation correction greatly surpass those expected to restore glucose homeostasis, and functional studies are ongoing to correlate pathophysiology to extent of mutation correction by base-editing.

Sickle Cell Disease 
Beam is pursuing two differentiated base editing approaches to treat hemoglobinopathies, BEAM-101 and BEAM-102

Beam Therapeutics -Third Quarter 2020 Financial Results

Cash Position: 
Cash, cash equivalents, and marketable securities were $202.2 million as of September 30, 2020. This cash balance does not include the proceeds of the October 2020 offering.

Net Loss: 
Net loss attributable to common stockholders was $34.5 million, or $0.69 per share, for the quarter ended September 30, 2020, compared to $22.3 million, or $3.31 per share, for the quarter ended September 30, 2019.

Reference
www.beamtx.com
https://investors.beamtx.com/node/6826/pdf

Comments

Popular posts from this blog

Carbohydrate Metabolism: MCQs and answers on Glycolysis & Gluconeogenesis

                                      MCQ on Glycolysis & Gluconeogenesis 1) Which of the following enzyme is not involved in galactose metabolism? a) Glucokinase b) Galactokinase c) Galactose-1-Phosphate Uridyl transferase d) UDP-Galactose 4- epimerase 2) Which of the following enzyme is defective in galactosemia (type I) - a fatal genetic disorder in infants? a) Glucokinase b) Galactokinase c) Galactose-1-Phosphate Uridyl transferase d) UDP-Galactose 4- epimerase 3) In the liver, the accumulation of which of the following metabolite attenuates the inhibitory of ATP on phosphofructokinase? a) Glucose-6-Phosphate b) Citrate c) Fructose-1,6-Bisphosphate d) Fructose-2,6-Bisphosphate 4) Mutation in which of the following enzymes leads to a glycogen storage disease known as "Tarui’s disease"? a) Glucokinase b) Phosphofructokinase c) Phosphoglucomutase d) Pyruvate Kinase 5) E...

MCQs and Answers on cultivation (culture/incubation), Isolation and Identification of microorganisms: Medical Microbiology

40 plus questions - Multiple Choice Questions on Classification, Culture, and Identification of the microorganisms 1. Which of the following microorganism has the cocci cell shapes and sizes arranged usually in tetrad structures? a)  Streptococcus pneumoniae b)  Staphylococcus aureus c)  Chlamydia trachomatis d)  Neisseria meningitidis 2. What are the different growth morphology and cell structures used for the classification of fungi? Select all the correct answers: a) Yeast b) Mold c) Mycelia d) Protozoa 3. Which of the following media is formulated with additional nutrients to support the growth of fastidious or nutritionally demanding bacteria that may not grow well on basic media? a) Differential media b) Enriched media c) Nutrient agar (media) d) Selective media 4. Which of the following metabolic characteristic is a distinguishing characteristic and identification of colonies of  E. coli ? a) Hydrogen sulfide formation b) Indole Formation c) Lactose fe...

Multiple Choice Questions (MCQs) on Diabetes Mellitus: Pathogenesis, Diagnosis and Treatment

                                        MCQs on Diabetes mellitus 1) Diabetes mellitus is a disorder characterized by hyperglycemia.  Which of the following is not the common characteristic features of type 2 diabetes mellitus ? a) Impaired insulin secretion b) Increased Insulin resistance  c) Diabetic ketoacidosis d) Excessive hepatic glucose production 2) Which of the following are the characteristic features of type 1 diabetes mellitus? a) Type 1 diabetes is caused by an absolute deficiency of insulin. b) Type 1 diabetes is associated with the autoimmune destruction of beta cells.  c) Patients with  uncontrolled type 1 diabetes present with diabetic ketoacidosis d) All of the above   3) Which of the following serum measurements are not used for the diagnosis of diabetes mellitus? a) Fasting blood glucose d) Postprandial blood glucose  c) Insulin ...