Rich Horgan believes that there’s an alternative path to drug development that can lower costs and the time it takes to bring drugs to market. He is trying to apply it to the field of rare and ultra-rare diseases.

Sarepta Therapeutics’ Elevidys is now the first FDA-approved gene therapy for Duchenne muscular dystrophy. Elevidys is an engineered version of a gene intended to restore function lost to the mutation at the root of the inherited muscle disease.

Private equity firm KKR teamed up with OMX Ventures to lead the seed investment in Replay, a company developing a suite of technologies that could overcome capacity limitations of adeno-associated viruses used for genetic medicines delivery. Duchenne muscular dystrophy is among the disease targets of the new startup.

Sarepta Therapeutics has more clinical data showing the safety and efficacy of its experimental gene therapy for Duchenne muscular dystrophy. A pivotal Phase 3 test is already underway and could post data next year but the company is also talking with regulators about the possibility of a submission under the accelerated approval pathway.

A Duchenne muscular dystrophy drug candidate from Italfarmaco Group has encouraging preliminary data from a pivotal study. The Milan, Italy-based pharmaceutical company says it now plans to meet with U.S. and European authorities about seeking regulatory approval for the small molecule.

Code Biotherapeutics uses synthetic DNA as the foundation for its genetic medicines, which the startup claims offer several key advantages compared to genetic medicines delivered via engineered viruses. The startup plans to use its new capital to develop lead programs in Duchenne muscular dystrophy and type 1 diabetes.

PepGen’s technology improves the delivery of a therapy to more tissue types and the clinical-stage biotech plans to use its IPO cash to continue developing its lead program for Duchenne muscular dystrophy. Meanwhile, eye products giant Bausch + Lomb returned to the public markets as a standalone company. Both the PepGen and Bausch + Lomb IPOs priced below their respective price ranges.

Pfizer now has the regulatory O.K. to resume pivotal tests of its gene therapy for Duchenne muscular dystrophy—but with new limitations and safety measures. The FDA had placed a clinical hold on the study late last year following a patient death.

The FDA has halted tests of a Pfizer gene therapy for Duchenne muscular dystrophy. The clinical hold is one of several placed on gene therapy tests in the past year due to safety concerns.

Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy posted encouraging results from a Phase 2 study enrolling 20 patients. But the FDA said a Phase 3 test is needed to support a regulatory submission, and the biotech is hunting for a pharmaceutical industry partner.

The FDA decision gives Sarepta Therapeutics its third approved drug for Duchenne muscular dystrophy. The accelerated approval requires the biotech to conduct additional clinical testing to confirm the drug’s benefit.

Despite a strongly critical complete response letter – which the FDA sent in August but did not make public until Tuesday – the drugmaker successfully appealed and won approval for the drug, Vyondys 53, in December, to the surprise of many.

An analyst wrote that the deal would create value for Sarepta, which has a gene therapy in Phase II development for Duchenne muscular dystrophy, SRP-9001, but lacks the resources to commercialize it abroad.

The FDA had previously rejected Sarepta’s application for Vyondys 53 in August, citing infections and kidney toxicity in preclinical studies. However, some analysts saw the move as a ‘slap on the wrist’ for the controversial approval of Sarepta’s first DMD drug.

In a note to investors, SVB Leerink analyst Joseph Schwartz suggested that the ‘questionable’ approval of Sarepta’s Exondys 51 may have played a role in the FDA’s decision to turn down golodirsen.

The decision stood in contrast with the agency’s highly controversial approval of Sarepta’s previous DMD drug, Exondys 51. The company’s shares fell sharply in after-hours trading.

Data from the drugmaker’s Phase Ib study showed patients responding, but an analyst wrote that it appears less potent and to have higher toxicity than that of its competitor, Sarepta Therapeutics.

An analyst wrote that the therapy’s combination of AAV delivery with a validated means of treating DMD could distinguish it from other gene therapies in what is already a crowded space.

After two painfully incremental drug approvals, the Duchenne muscular dystrophy field is still searching for a therapy that can substantially improve patients’ lives and lifespan. Sounds like a job for CRISPR/Cas9.

Emflaza, the brand name for deflazacort, has never been approved for sale in the United States, but the steroid has been sold for decades in other countries — at much lower prices

Marathon Pharmaceuticals is doing some backtracking after the public, the media and now Bernie Sanders objected to the list price of its upcycled corticosteroid for Duchenne muscular dystrophy.

The FDA has given the green light to Emflaza (deflazacort), a new drug for Duchenne muscular dystrophy. Announced Thursday, the news is bittersweet. Deflazacort is a corticosteroid, a class of drugs that doctors have used off-label for many years.

Exondys 51 doesn’t cure Duchenne muscular dystrophy and will only help a minority of patients.