Innovation holds the key in the ever-evolving biotech sector, and most companies in this sector are striving to develop a drug or treatment using a breakthrough technology.
Biotech companies with promising gene therapies in their portfolio/pipeline have garnered investors’ attention in 2024. Interest in this space renewed following the FDA approval of a few gene therapies toward the end of 2023.
Consequently, some pharma/biotech bigwigs scurried for a lucrative catch in the space, either through licensing deals or acquisitions.
What is Gene Therapy?
Gene therapy enables the modification of a patient’s gene to treat or cure the underlying disease. The treatment involves either replacing a disease-causing gene with a healthy copy of the gene or disabling a disease-causing gene that is not functioning properly. A new or modified gene can also be introduced into the patient’s body to help treat a disease.
The therapy takes a different approach from traditional drug therapies as it can treat the underlying cause and not just the symptoms.
Given the treatment's potential, gene therapy products are being evaluated to treat diseases, including cancer, genetic diseases and infectious diseases. However, the uncertainty regarding the uptake of these therapies, given the high prices of these treatments and their side effects, can deter investors. Nevertheless, this space remains in the spotlight in a volatile biotech sector, considering the need for innovative treatments.
Here, we pick three gene-therapy stocks, namely Sarepta Therapeutics SRPT, CRISPR Therapeutics CRSP and Voyager Therapeutics VYGR, which are likely to perform well in 2025 on the strength of their strong portfolio/pipeline.
Our Gene-Therapy Stock Picks
Sarepta’s portfolio received a significant boost with the FDA approval for Elevidys — the first-ever gene therapy for the Duchenne muscular dystrophy (DMD) indication. Since its launch, the gene therapy has demonstrated blockbuster potential. In June 2024, the FDA granted label expansion approval to Elevidys to treat all DMD patients aged four years and older.
SRPT is also working to add new gene therapy treatments to its portfolio. It is developing gene therapy programs for Limb-girdle muscular dystrophy (LGMD). It plans to submit a regulatory filing with the FDA in mid-2025 for its LGMD candidate, SRP-9003. The company is also developing potential therapeutic candidates for other neuromuscular and central nervous system disorders.
Apart from Elevidys, Sarepta has three other therapies — Exondys 51, Vyondys 53 and Amondys 45 — in its commercial portfolio targeting the DMD patient population.
Sarepta has put up a strong performance in 2024, with shares surging 31.5%. Positive regulatory updates and good pipeline progress should help the stock gain further. SRPT currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Sarepta Therapeutics, Inc. Price and Consensus
Sarepta Therapeutics, Inc. Price and Consensus
Sarepta Therapeutics, Inc. price-consensus-chart | Sarepta Therapeutics, Inc. Quote
CRISPR Therapeutics is a leading gene editing company focused on developing CRISPR/Cas9-based therapeutics, which have huge potential. The company received a significant boost with the approval of a CRISPR-based therapy, Casgevy (exagamglogene autotemcel [exacel]), for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in patients aged 12 years and older. It has partnered with Vertex Pharmaceuticals for the development and commercialization of Casgevy. The initial uptake of the therapy is encouraging.
CRSP is pursuing the development of its candidates to create novel CAR-T cell therapies. Management is developing two next-generation CAR-T therapy candidates — CTX112 (targeting CD19-positive B-cell malignancies) and CTX131 (targeting relapsed or refractory solid tumors) — in separate phase I/II studies. While CTX112 is being developed for oncology and autoimmune indications, CTX131 is being developed for solid tumors and hematologic malignancies.
CRSP currently carries a Zacks Rank #2. Loss per share estimates for 2024 and 2025 have narrowed 7 cents and 6 cents, respectively, to $5.13 and $5.17 in the past 30 days. The current average target price of $81 for CRSP represents an upside of 85.02%.
CRISPR Therapeutics AG Price and Consensus
CRISPR Therapeutics AG Price and Consensus
CRISPR Therapeutics AG price-consensus-chart | CRISPR Therapeutics AG Quote
Voyager’s wholly-owned, prioritized pipeline programs include an anti-tau antibody for Alzheimer’s disease (AD); a superoxide dismutase 1, or SOD1, silencing gene therapy for amyotrophic lateral sclerosis (ALS); and a tau silencing gene therapy for AD. The company has made good pipeline progress so far.
VYGR’s lead product candidate is VY7523, an anti-tau antibody for AD. Enrollment and dosing have been completed in the single ascending dose clinical study, with top-line safety and pharmacokinetic data expected in the first half of 2025. An increasing body of clinical evidence implicates tau as a critical target in AD.
The company recently selected a lead development candidate, VY1706, for its tau silencing gene therapy program in AD and anticipates filing an investigational new drug (IND) application with the FDA and a clinical trial application (CTA) with Health Canada for the candidate in 2026.
VY9323 is currently in preclinical development for ALS. VYGR expects to submit an IND application to the FDA and CTA to Health Canada for this program in mid-2025.
Voyager Therapeutics, Inc. Price and Consensus
Voyager Therapeutics, Inc. Price and Consensus
Voyager Therapeutics, Inc. price-consensus-chart | Voyager Therapeutics, Inc. Quote
VYGR also collaborated with and has license agreements with bigwigs AstraZeneca and Novartis.
Shares of the company have risen 4% in three months. VYGR currently carries a Zacks Rank #3 (Hold). Loss per share estimates for the company have narrowed 15 cents for 2024 and 24 cents for 2025 in the past 30 days. The current average target price of $17.1 for VYGR represents an upside of 172.73%.
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