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 hydroxylase (PAH) gene that cause PKU.  

PKU is a rare, inherited metabolic disorder that results in toxic accumulation of phenylalanine (Phe), leading to serious neurologic and neurocognitive impairments and lifelong dietary management. By correcting mutations in the PAH gene, BEAM-304 aims to reduce toxic Phe to within recommended guidelines while enabling normalisation of diet and freedom from medical food. 

“In 2025, we established base editing as a best-in-class technology for genetic medicine, with positive proof-of-concept data and regulatory and clinical development paths to approval across multiple high-value programmes,” said John Evans, CEO of Beam Therapeutics.  

“The announcement of BEAM-304 for PKU marks an important expansion of our pipeline and exemplifies the power and scalability of our platform.  

“By combining our clinically validated base editing technology with our internally discovered and optimised lipid nanoparticle (LNP) delivery, we have the potential to bring forward one-time, durable treatments for the vast majority of PKU patients.”

Beam looks to launch ‘best-in-class’ sickle cell disease treatment
 

The programme expansion comes as Beam announced a $500 million strategic financing agreement with investment firm Sixth Street. 

The financing will support the potential launch of its ristoglogene autogetemcel (risto-cel) in sickle cell disease (SCD).

“This strategic financing provides Beam with significant flexibility and long-term, non-dilutive capital to support the anticipated commercial launch and subsequent revenue generation for risto-cel, which we believe has the potential to be a best-in-class, one-time treatment for sickle cell disease,” said Sravan Emany, Chief Financial Officer of Beam Therapeutics. 

“By underwriting the commercialisation costs of risto-cel, this facility secures this important, high-value franchise and also enhances our ability to direct our capital to the growth of our pipeline and execute our long-term vision for precision genetic medicines.”