Beam Therapeutics develops precision genetic medicines using a technique called base editing, which allows for precise modifications to the genetic code to potentially correct mutations that lead to serious diseases. Their primary focus is on treating genetic disorders, such as sickle cell disease, with the aim of providing lifelong cures rather than temporary solutions. The company invests significantly in research and development to create new therapies and generates revenue through partnerships, licensing agreements, and future commercialization of their treatments. Beam Therapeutics distinguishes itself from competitors by its commitment to rigorous scientific research and a talented team of experts dedicated to advancing genetic medicine. The ultimate goal of the company is to deliver lasting cures for patients suffering from genetic conditions.

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Beam Therapeutics

CAMBRIDGE, Mass., Dec. 06, 2024 (GLOBE NEWSWIRE) - Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the appointment of Sravan K. Emany as chief financial officer (CFO), effective December 19, 2024.

Beam Therapeutics Announces Appointment of Sravan Emany as Chief Financial Officer

CAMBRIDGE, Mass., July 15, 2024 (GLOBE NEWSWIRE) - Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that its chief financial officer, Terry-Ann Burrell, will be leaving the company effective August 9, 2024 to pursue a new opportunity as vice chairman of investment banking at JPMorgan Chase.

Beam Therapeutics Chief Financial Officer Terry-Ann Burrell to Retire

Rare Diseases – Curing The IncurableWhile not entirely solved, a lot of major deadly illnesses that have plagued humanity have become more manageable over the last century, from bacterial (antibiotics) and viral (vaccines) infections to diabetes (insulin).There is, however, a wide array of rare diseases which are still mostly incurable. Most of the time, this is because these diseases are not caused by an external threat, but by a biological dysfunction inside the very cells of the patient. This is quickly changing, thanks to progress in biosciences.On one hand, with the human genome sequenced and much more advanced analytical methods, scientists can finally understand what are the root causes of rare diseases. Even AI is now helping interpret the data and provide new insights .On the other hand, new technologies like gene editing open the possibility of fixing deficient cells or organs, restoring normal function.Rare diseases each affect less than 1 in 2,000 people, with more than 6000 different rare diseases. Despite their individual rarity, together, they affect around 4% of the population, with as many as 30 million people in just Europe .3/4 of rare diseases affect children, 72% are genetic in origin, and it takes on average 5 years for rare disease patients to get a diagnosis.CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a recently discovered tool for genetic editing. It allows for very precise and directed gene editing, and its discoverers have won the 2020 Nobel Prize .What sets CRISPR Therapeutics apart is the all-star team of founders, including Dr

10 Rare Diseases Specialist Stocks

Beam Therapeutics is developing BEAM-101 for the SCD indication in early-stage studies.

Vertex (VRTX), CRSP's Casgevy Gets EU Nod for Two Blood Disorders

Beam Therapeutics appoints biotech executive Christi Shaw to its board of directors.

Beam Therapeutics Appoints Biotech Executive Christi Shaw to its Board of Directors

Eli Lilly is acquiring Beam Therapeutics' opt-in rights to three Verve Therapeutics gene-editing therapies for cardiovascular conditions.

Eli Lilly Strikes $250M Deal to Add Gene-Editing Medicines for Cardio Conditions

Beam Therapeutics to sell rights in Verve licensing deal to Lilly for $250 million upfront.

Beam Therapeutics to Sell Rights in Verve Licensing Deal to Lilly for $250 Million Upfront

A Crippling And Painful DiseaseSickle Cell Disease (SCD) is a blood disease caused by a genetic mutation. This mutation creates abnormal hemoglobin, the protein carrying oxygen in the blood's red cells.As a result, red cells are shaped like sickles and tend to get stuck in blood vessels, causing reduced blood flow and obstruction. Such obstruction can cause extreme pain, swelling, vision problems, and sensitivity to infections.This also causes red cells to die off in just 10 to 20 days instead of the normal 120 days, causing anemia in the patients.This is a disease affecting more than 20 million people worldwide, of which 100,000 are in the USA.It also disproportionately affects people of African ancestry, with 1 in 13 Black or African American babies being born with sickle cell trait, and 1 in every 365 Black or African American babies being born with sickle cell disease.Because the disease affects each individual blood cell produced by the body, efficient treatments have long been impossible to develop, with most healthcare limited to reducing the severity or consequences of the symptoms.Red cells are also constantly produced and recycled in the body, so ideally, a cure would repair the body's capacity to create functional/normal red cells.The Gene Therapy MiracleWhat made SCD so difficult to cure, its genetic origin, is also what makes it uniquely fit for the novel tools of gene therapy, especially gene editing. The mutation affects only one gene, and in most cases, only a single nucleotide (one letter of the genetic code).This means that if we could modify that one letter in the patient's DNA, we could cure the disease entirely for life.Previous generations of gene therapies struggled to be precise enough to provide a cure for SCD. But with the emergence of CRISPR technology, able to precisely target and edit genes one nucleotide at a time, this might become possible soon.Many companies are working on this technology, with SCD the prime focus of many of them.Gene Editing Companies Working On An SCD CureCRISPR TherapeuticsCRISPR Therapeutics was founded by CRISPR Cas9 co-discoverer and 2020's Nobel Prize winner Emmanuel Charpentier. The company focuses on applying to human medicine the CRISPR Cas9 system.CRISPR Therapeutics is working in close collaboration with larger biotech Vertex to develop therapies for blood diseases (Beta-thalassemia and SCD), as well as a potential cure for Type-1 diabetes.For curing both Beta-thalassemia  SCD, CRISPR Therapeutics is looking to replace the deficient hemoglobin with fetal hemoglobin (HbF), which is naturally present in all people before birth and with a higher affinity for oxygen than adult hemoglobin.The cure could work for both, because SCD patients have the wrong type of hemoglobin, while beta-thalassemia do not have enough hemoglobin

How Crispr Companies Target Sickle Cell Anemia

Beam in 2020 disclosed its plans to invest $83 million in a manufacturing facility in RTP.

Beam Therapeutics, with RTP facility, to lay off 100

In addition to shelving some programs, Beam said Thursday that it will lay off about 100 employees, representing 20% of the biotech's staff.

Base-Editing Biotech Beam Restructures, Narrowing Pipeline & Cutting 20% of Staff - MedCity News

Develops precision genetic medicines using base editing

Beam Therapeutics

Develops precision genetic medicines using base editing

Beam Therapeutics

Develops precision genetic medicines using base editing

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