Beam Therapeutics will present initial data from the ongoing Phase I/II clinical trial evaluating BEAM-301 for the treatment of glycogen storage disease type Ia (GSDIa) at the 33rd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). BEAM-301 is a liver-targeting lipid-nanoparticle (LNP) formulation… Read More
The US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for Beam Therapeutics’ BEAM-304 for the treatment of phenylketonuria (PKU). PKU is a rare, inherited metabolic disorder that results in toxic accumulation of phenylalanine (Phe), leading to serious neurologic and neurocognitive impairments and requires strict,… Read More
Biotechnology company Beam Therapeutics has reported encouraging data for its treatment for the genetic disease Alpha-1 Antitrypsin Deficiency (AATD). Beam announced BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation designed to directly correct the underlying genetic mutation that causes AATD, has shown a well-tolerated safety profile and efficacy in a… Read More
Biotechnology company Beam Therapeutics is set to expand its liver-targeted genetic disease franchise for the treatment of phenylketonuria (PKU). Beam’s lead programme, BEAM-304, leverages the company’s proprietary and clinically validated base editing technology and lipid nanoparticle (LNP) delivery capabilities to directly and durably correct mutations in the phenylalanine… Read More
Biotechnology company Beam Therapeutics says initial data from its Phase I/II trial establish clinical proof-of-concept for BEAM-302 as a potential treatment for alpha-1 antitrypsin deficiency (AATD) and for in vivo base editing. Preliminary results from the first three single-ascending dose cohorts demonstrated that BEAM-302 was… Read More
Researchers have developed a gene editing therapy that could potentially treat Stargardt disease, the most common form of inherited macular degeneration. The study, published in Nature Medicine, demonstrates the therapeutic use of a precision gene editing technology called base editing. … Read More
The first quadruplex-edited, allogeneic CAR-T cell therapy candidate has entered clinical trials in the US. The first patient has been treated with Beam Therapeutics’ BEAM-201 as part of a Phase I/II clinical study for the treatment of relapsed/refractory T-cell acute lymphoblastic leukaemia/T-cell lymphoblastic lymphoma (T-ALL/T-LL). Read More
