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Gene Therapy

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7 ARTICLES

2mo ago

FDA Places Clinical Hold on REGENXBIO's RGX‑121 After Spine MRI Findings

Regulators have ordered additional imaging and longer follow‑up to determine whether the asymptomatic spinal nodules found in trial participants are linked to the AAV gene therapy which has paused near‑term approval plans.

7 ARTICLES

4mo ago

FDA Agrees Three‑Year Phase I/II Data Can Support uniQure’s AMT‑130 Accelerated BLA

19 ARTICLES

6mo ago

FDA Approves First Gene Therapy for Inherited Hearing Loss

10 ARTICLES

7mo ago

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

8 ARTICLES

7mo ago

Sarepta Reports Early siRNA Data Showing Muscle Delivery as Shares Surge

7 ARTICLES

7mo ago

Plaintiff Firms Intensify Biotech Securities Suits as Key Deadlines Near for Nektar, uniQure and REGENXBIO

3 ARTICLES

7mo ago

Taysha Advances Rett Gene Therapy Program With Pivotal Dosing, FDA Alignment and Q2 Data Timeline

3 ARTICLES

8mo ago

uniQure Investors Face April 13 Deadline in Securities Class Action Over FDA Setback

3 ARTICLES

8mo ago

uniQure Soars 36% After FDA Biologics Chief Vinay Prasad Announces April Exit

4 ARTICLES

9mo ago

FDA Rejects Regenxbio’s Hunter Syndrome Gene Therapy Application

4 ARTICLES

9mo ago

Lilly Strikes Up to $1.12 Billion Pact With Seamless Therapeutics for Hearing‑Loss Gene Editing

5 ARTICLES

last yr.

Kriya Nets $320 Million Series D as Odyssey Raises $213 Million and Amgen Retools Research Leadership

15 ARTICLES

last yr.

Regulators Halt Elevidys Gene Therapy Worldwide After Safety Failures

7 ARTICLES

last yr.

Sarepta Defies FDA, Keeps Duchenne Gene Therapy on the Market

16 ARTICLES

last yr.

Sarepta Shares Plummet on FDA Review of Elevidys Following Third Patient Death

3 ARTICLES

last yr.

Eli Lilly to Acquire Verve Therapeutics as Gene-Editing Trial Begins

8 ARTICLES

last yr.

Sarepta Halts Elevidys Shipments for Non-Ambulatory Patients After Second Fatal Liver Failure

Gene Therapy

FDA Places Clinical Hold on REGENXBIO's RGX‑121 After Spine MRI Findings

FDA Agrees Three‑Year Phase I/II Data Can Support uniQure’s AMT‑130 Accelerated BLA

FDA Approves First Gene Therapy for Inherited Hearing Loss

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

Sarepta Reports Early siRNA Data Showing Muscle Delivery as Shares Surge

Plaintiff Firms Intensify Biotech Securities Suits as Key Deadlines Near for Nektar, uniQure and REGENXBIO

Taysha Advances Rett Gene Therapy Program With Pivotal Dosing, FDA Alignment and Q2 Data Timeline

uniQure Investors Face April 13 Deadline in Securities Class Action Over FDA Setback

uniQure Soars 36% After FDA Biologics Chief Vinay Prasad Announces April Exit

FDA Rejects Regenxbio’s Hunter Syndrome Gene Therapy Application

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Gene Therapy

Lilly Strikes Up to $1.12 Billion Pact With Seamless Therapeutics for Hearing‑Loss Gene Editing

Lilly Licenses MeiraGTx Eye Gene Therapy in Deal Worth Up to $475 Million

Sarepta Shares Plunge After ESSENCE Phase 3 Miss in Duchenne Trial

FDA Setback Puts uniQure’s Huntington’s Gene Therapy Filing in Doubt

Kriya Nets $320 Million Series D as Odyssey Raises $213 Million and Amgen Retools Research Leadership

Regulators Halt Elevidys Gene Therapy Worldwide After Safety Failures

Sarepta Defies FDA, Keeps Duchenne Gene Therapy on the Market

Sarepta Shares Plummet on FDA Review of Elevidys Following Third Patient Death

Eli Lilly to Acquire Verve Therapeutics as Gene-Editing Trial Begins

Sarepta Halts Elevidys Shipments for Non-Ambulatory Patients After Second Fatal Liver Failure

Never miss stories about

Gene Therapy