Solid Biosciences Inc. SLDB announced that the FDA has cleared its investigational new drug (IND) application to begin clinical studies on its novel AAV-based gene therapy candidate, SGT-212, for the treatment of Friedreich’s ataxia (FA).

Per the company, SGT-212 is the only full-length frataxin replacement gene therapy candidate that targets the cardiac manifestations of FA.

SGT-212 is being developed to treat the neurologic and systemic clinical manifestations of FA to address the full spectrum of disease progression.

Shares of the company were up 17.4% in after-hours trading on Jan. 7 following the announcement of the news. The stock was also up in pre-market trading on Jan. 8.

In the past year, shares of Solid Biosciences have plunged 51.4% compared with the industry’s decline of 15.7%.

Image Source: Zacks Investment Research

More on SLDB's FA Gene Therapy Candidate

The above IND was backed by robust preclinical data on SGT-212.

SLDB plans to initiate a phase Ib study SGT-212 for treating FA in the second half of 2025.

The first-in-human, open-label, dose-finding study will evaluate the safety and tolerability of systemic intravenous infusion and direct intradentate nuclei (IDN) infusion of SGT-212 in non-ambulatory and ambulatory adult patients with FA.

Per management, the dual route of administration helps in the direct delivery of SGT-212 to the heart and cerebellum to potentially treat the most significant symptoms of FA.

The gene therapy candidate provides a differentiated approach to treat the underlying disease.

Other Players in the FA Market

We note that FA is a rare disease. Hence, a few companies have developed or are developing a treatment for the disease.

Drug giant, Biogen BIIB currently markets Skyclarys, which is the first approved treatment for FA. The FDA approved the drug in 2023, while it was approved in Europe in February 2024.

The acquisition of Reata Pharmaceuticals in September 2023 added this rare disease drug to Biogen’s portfolio.

Per BIIB, the launch of Skyclarys is progressing well in the United States and the European Union as the drug is witnessing increased demand globally.

Clinical-stage company Larimar Therapeutics LRMR is developing its lead pipeline candidate nomlabofusp (CTI-1601) as a potential treatment for FA.

Larimar Therapeutics plans to file a biologics license application seeking approval of nomlabofusp for treating FA in the second half of 2025.

LRMR believes that nomlabofusp has the potential to be the first frataxin protein replacement therapy for patients with FA.

SLDB's Zacks Rank & Stock to Consider

Solid Biosciences currently carries a Zacks Rank #3 (Hold).

A better-ranked stock in the biotech sector is Puma Biotechnology, Inc. PBYI, sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for Puma Biotechnology’s 2025 earnings per share have increased from 42 cents to 54 cents. In the past year, shares of PBYI have declined 21.7%.

PBYI’s earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 32.78%.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report

Biogen Inc. (BIIB) : Free Stock Analysis Report

Puma Biotechnology, Inc. (PBYI) : Free Stock Analysis Report

Solid Biosciences Inc. (SLDB) : Free Stock Analysis Report

Larimar Therapeutics, Inc. (LRMR) : Free Stock Analysis Report

To read this article on Zacks.com click here.

Zacks Investment Research