Shares of Beam Therapeutics BEAM rose 5% on Thursday after the FDA cleared the investigational new drug (IND) application for BEAM-304, the company’s investigational gene-editing therapy for the treatment of phenylketonuria (PKU). The clearance marks an important milestone for Beam Therapeutics as it expands its liver-targeted genetic disease franchise.
PKU is a rare inherited metabolic disorder characterized by the inability to properly break down phenylalanine (Phe), resulting in its toxic buildup in the body. If left untreated, elevated Phe levels can lead to serious neurological and cognitive complications, requiring patients to adhere to strict lifelong dietary restrictions.The disease affects approximately 20,000 people in the United States and is typically diagnosed through newborn screening. Currently, no approved curative treatments exist for PKU.
Year to date, the stock has risen 23.2% against the industry’s 1% decline.
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More on Beam Therapeutics’ BEAM-304
BEAM-304 is a liver-targeted, lipid nanoparticle (LNP)-based gene-editing therapy designed to correct disease-causing mutations in the phenylalanine hydroxylase (PAH) gene responsible for PKU. By restoring PAH function, the therapy aims to lower toxic Phe levels, potentially allowing patients to maintain a normal diet without relying on specialized medical foods.
The candidate is being developed using an innovative approach that enables efficient evaluation of multiple mutation-specific base editors within a single clinical program through in vivo delivery. This strategy aligns with the FDA’s efforts to accelerate the development of genome-editing treatments for serious genetic diseases.
In preclinical studies, BEAM-304 demonstrated robust on-target liver editing and normalization of Phe levels in PKU mouse models at clinically relevant doses, supporting its potential as a disease-modifying therapy.
Beam plans to evaluate BEAM-304 in a phase I/II open-label clinical study designed to initially assess the safety, tolerability, blood Phe reduction and the potential for dietary liberalization of BEAM-304 in PKU patients with the R408W mutation. The therapy will be administered as an intravenous infusion. The program will then expand to evaluate a base editor targeting a second mutation, with the aim of establishing clinical proof of concept for base editing in PKU.
BEAM’s Zacks Rank & Stocks to Consider
Beam Therapeutics currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Indivior Pharmaceuticals INDV, Liquidia Corporation LQDA and Immunocore IMCR, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, earnings per share estimates for Indivior Pharmaceuticals remained unchanged at $4.05 for 2026 and $4.27 for 2027. INDV shares have risen 6.7% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 65.44%.
Over the past 30 days, estimates for Liquidia’s earnings per share remained unchanged at $2.97 for 2026 and $4.81 for 2027. LQDA shares have surged 106.1% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
Over the past 30 days, earnings per share estimates for Immunocore’s 2026 were unchanged at 6 cents for 2026 and 87 cents for 2027. IMCR shares have lost 17.6% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 46.66%.
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