With the progression of modern medicine, and the continuous discoveries in the realm of gene editing, investing early in the technology could be a game changer for long-term returns if selecting the best gene editing stocks to buy. That's because unlike more standard therapeutics such as antibiotics, antivirals and vaccines gene editing is a permanent solution most of the time. It changes the base instructions of the human body for how the body is maintained and protected. CRISPR Therapeutics (CRSP) When it ...

Core Insights - Beam Therapeutics is advancing its investigational therapy BEAM-302 for alpha-1 antitrypsin deficiency (AATD) with the first patient dosed in a phase I/II study [10] - The company has received clinical trial authorization from the UK Medicines and Healthcare Products Regulatory Agency for BEAM-302 in March 2024 [2] - Beam is also developing BEAM-301 for glycogen storage disease 1a, with an investigational new drug application expected to be filed soon [3] Company Developments - The open-label, dose-escalation study for BEAM-302 aims to assess safety, pharmacodynamics, pharmacokinetics, and efficacy [1] - The BEACON study has completed dosing and engraftment for three patients, with data from multiple patients expected in the second half of 2024 [4] - Beam's leading ex-vivo genome-editing candidate, BEAM-101, is in the BEACON study for sickle cell disease, which could enhance the company's future prospects if successful [11][12] Market Performance - Beam Therapeutics shares have declined 8.7% year to date, compared to a 4.6% decrease in the industry [11] - The company currently holds a Zacks Rank of 3 (Hold) [13]

Company Overview - Beam Therapeutics is a biotechnology company focused on developing precision genetic medicines through base editing technology [6][8] - The company aims to establish a fully integrated platform for precision genetic medicines, including gene editing and delivery technologies, and is building internal manufacturing capabilities [6][14] Product Development - The first patient has been treated with BEAM-302, an investigational in vivo base editing medicine targeting severe alpha-1 antitrypsin deficiency (AATD) [8][4] - BEAM-302 is designed to correct the PiZ mutation associated with AATD, which affects liver and lung function [9][12] - The clinical trial for BEAM-302 is a Phase 1/2 open-label, dose-escalation study evaluating safety, pharmacodynamics, pharmacokinetics, and efficacy [12] Disease Context - AATD is an inherited genetic disorder that can lead to early onset emphysema, liver disease, and other complications [13] - Approximately 100,000 individuals in the U.S. have the PiZZ genotype, but only 10-15% are diagnosed [2][13] - Current treatments, such as intravenous AAT protein replacement, do not prevent ongoing lung function decline [2] Therapeutic Potential - BEAM-302 aims to provide a one-time therapy that corrects the genetic mutation, potentially reducing liver toxicity and increasing functional AAT protein levels [9][4] - The correction of the PiZ mutation is expected to be durable based on preclinical evidence, addressing the underlying pathophysiology of both liver and lung diseases [9][4]

CAMBRIDGE, Mass., June 26, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the first patient was treated with BEAM-302, an investigational in vivo base editing medicine designed to precisely correct the underlying cause of severe alpha-1 antitrypsin deficiency (AATD), that is currently being evaluated in a Phase 1/2 clinical trial. "Treating the first patient with BEAM-302 is an important mil ...

Beam designed its automated CD34+ HSPC process for manufacturing of BEAM-101 to have the flexibility for a wide range of patient-starting material and variable cell numbers, while maintaining robust cell yield and high drug product quality suitable for use in the BEACON Phase 1/2 clinical trial. Today's data highlight the following: About Beam Therapeutics | --- | |---------------------| | | | Contacts: | | Investors: | | Holly Manning | | Beam Therapeutics | | hmanning@beamtx.com | | Media: | | Dan Budwick ...

"The manufacturing of autologous cell and gene therapies, particularly for sickle cell disease, is complex and has significant implications for product quality as well as the patient experience," said Giuseppe Ciaramella, Ph.D., president of Beam. "The data presented today at EHA demonstrate that our optimized process for the manufacturing of BEAM-101, which integrates key technologies and automation improvements, has achieved reproducible and robust product yields and viability that meet demanding high-qua ...

Will the recent positive trend continue leading up to its next earnings release, or is Beam Therapeutics due for a pullback? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at its most recent earnings report in order to get a better handle on the important drivers. Total revenues, comprising license and collaboration revenues, came in at $7.4 million in the first quarter compared with $24.2 million in the year-ago period. The top line missed the Zacks Consensu ...

CAMBRIDGE, Mass., May 14, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will report data highlighting the robust, closed and automated manufacturing process for BEAM-101, as deployed in the BEACON Phase 1/2 clinical trial, in a poster presentation at the European Hematology Association (EHA) Hybrid Congress, hosted virtually and in person in Madrid, Spain on June 13-16, 202 ...

CAMBRIDGE, Mass., May 14, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will report data highlighting the robust, closed and automated manufacturing process for BEAM-101, as deployed in the BEACON Phase 1/2 clinical trial, in a poster presentation at the European Hematology Association (EHA) Hybrid Congress, hosted virtually and in person in Madrid, Spain on June 13-16, 202 ...

Beam Therapeutics Inc. (BEAM) incurred a loss of $1.21 per share in the first quarter of 2024, narrower than the Zacks Consensus Estimate of a loss of $1.45. The company had recorded a loss of $1.33 per share in the year-ago quarter.Total revenues, comprising license and collaboration revenues, came in at $7.4 million in the first quarter compared with $24.2 million in the year-ago period. The top line missed the Zacks Consensus Estimate of $14 million.Quarter in DetailResearch and development expenses amou ...