Monogenic Rare Neuro Gene Therapies

	A	B	C	D	E	F	G	H	I	J	K	L	M	N	O
1	Order	Drug	Gene	Life-limiting?	US est. Pop	aka	Company	IND	Dose	Approval	Route	US Age/Sex	Link	Phase	Other
2	1	Zolgensma	SMN1	Yes		SMA all types	Novartis (Avexis)	6/23/2017	7/20/2017	5/24/2019	IV
3	2	Zynteglo	SMN1	Yes		SMA t2 and t3	Bluebird/Spark	6/26/2018	10/31/2018	5/27/2022	IT
4	3	Upstaza	DDC	No?	?	AADC Deficiency	PTC Therapeutics			7/18/2022 (EMA)	Putamen	18 months+	https://aadcnews.com/ptc-aadc/
5	4	PBGM01	GLB1	Yes		GM1 Gangliosidosis	Passage Bio	1/4/2021		Not yet	ICM		Passage Bio Announces Promising Interim Clinical Data from First Eight Patients with GM1 Gangliosidosis in Imagine-1 Study	1/2
6	5	NGN-101	CLN5	Yes	Tiny	Batten	Neurogene	9/1/2021	1/1/2022	Not Yet	intracisternal		https://www.cgtlive.com/view/neurogene-clinical-trial-seeks-assess-cln5-batten-disease-gene-therapy-ngn-101	1/2
7	6	Melpida	AP4M1	Yes	Tiny	SPG50		12/30/21	3/24/2022	Not Yet	IT		https://toronto.ctvnews.ca/toronto-boy-with-rare-genetic-disorder-begins-treatment-after-family-raises-us-3-million-1.6027732		Canada
8	7	TSHA-102	MECP2	No	30,000 per Jag	Rett	Taysha	8/14/23	1/10/2024	Not yet	IT	3-8 Females	https://ir.tayshagtx.com/node/9036/pdf	1/2	Axovant Gene Therapies, Biogen, and Voyager Therapeutics
9	8	GS-100	NGLY1	Maybe	Tiny		Grace Science	11/7/23		Not yet	ICV	2-18 both	https://www.biospace.com/article/releases/grace-science-announces-fda-clearance-of-investigational-new-drug-ind-application-to-initiate-a-phase-1-2-3-trial-for-the-treatment-of-ngly1-deficiency-with-gs-100-an-aav9-ngly1-gene-therapy/	1/2/3
10	9	JAG201	SHANK3	No	?	PMS	Jaguar	1/31/24	2H24	Not yet	ICV	Adults both	https://pmsf.org/breaking-news-from-jaguar-gene-therapy/	1
11	10	ETX101	SCN1A	Yes	?	Dravet	Encoded	2/6/24	1H24	Not yet		0-3 (US) 3-7 (Aus)	https://encoded.com/press-releases/encoded-therapeutics-announces-us-ind-clearance-and-australian-cta-approval-for-dravet-syndrome-gene-therapy-candidate-etx101/	1/2
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